CATALINA FOOTHILLS, Ariz. (KGUN) — After 37 years and against all odds, one woman with lysosomal acid lipase deficiency (LAL-D) found hope and healing through a medication called Kanuma The Namazifard family moved to Tucson in 1987 from Kuwait seeking treatment for two sisters with a rare genetic disease involving the liver After decades of endless doctor appointments and no real improvements in health lives with the same rare genetic disease but has yet to start the treatment "I never expected in my lifetime that a treatment would be available," Shaida said Shaida was diagnosed with LAL-D at 17 years old alongside her sister Shaima who was just four years old causing fat to build up in the body's organs and tissues which could lead to liver disease and high cholesterol "All I knew and remember is that I was different," Shaima said the two sisters were interviewed by KGUN 9 because of their rare condition: At the time, they were prescribed Lovastatin a treatment for high cholesterol and triglyceride levels no treatment for their condition was available "I've been on medication for 35 years," Shaima said A pharmaceutical company, Alexion, created Kanuma for LAL-D. It was FDA-approved in 2015 and released in 2016 "I have been mentioning this to every specialist every primary I have seen for the last 37 years," Shaida said It wasn't until late 2023 that Shaida met with Dr Ebrahimi was studying for her board exams around the same time she met Shaida and learned about Kanuma "The medication is basically giving you the enzyme and is a lifetime treatment," Dr "I'm four months into the treatment and it looks like the body has reset itself," Shaida said in July Shaida invited me to one of her infusion appointments on July 10 The appointments typically last two to three hours and she takes the day off work to recover she would get the infusion through her peripheral veins making the infusion process longer and painful she opted to get a port and says she has no issues with it "I repeated the lab and her liver enzyme for the first time in her life came back normal," said Dr Ebrahimi's goal is to spread awareness about how good communication between a doctor and patient is crucial to finding proper treatment for an illness Shaima is currently looking into getting the same treatment Shaida says she found a group on Facebook with only 113 people from all over the world sharing their LAL-D diagnosis She encourages anyone with the condition to join it Report a typo The Financial Express Happy Kanuma 2025 Wishes and Quotes: Kanuma It is dedicated to honoring the animals that assist in crop cultivation these animals are bathed in wells or ponds and they are treated to special meals and given complete rest The celebration acknowledges that the animals play a vital role in the farmers’ work birds that help control pests in crops are also revered grains are often hung on doorsteps in their honor While the Mukkanuma festival takes place on the fourth day the festivities of Sankranti largely wind down with Kanuma This day serves as a time to express gratitude to the farmers who provide us with food Celebrate Kanuma with gratitude for the blessings of nature and livestock Wishing you love and abundance today and always Let us honour the traditions of our ancestors and the bond we share with animals and the earth May your Kanuma be as special as the love and gratitude you show to your cherished livestock and family Kanuma is a reminder of our roots and our bond with nature Wishing you a Kanuma that is as bright as the festive lights May the blessings of Kanuma bring joy to your heart and success to your efforts Let this day remind us to cherish the gifts of nature and family and filled with gratitude for the simple joys of life Kanuma reminds us to cherish the gifts of the earth and the love of our family May this festival bring you lasting happiness and success and the abundance of nature fill your heart and home with blessings I wish you happiness as endless as the skies and blessings as rich as the fields Let’s celebrate Kanuma by honouring our roots and traditions May the light of Kanuma brighten your life and the blessings of this festival make your days as golden as the harvest fields Bain Capital has requested permission from CCI to purchase a major portion of Manappuram Finance through a multi-layered deal worth Rs 4,385 crore No competition concerns have been raised as there are no overlaps between the businesses of the two companies the Gomahotsavam has been celebrated with religious fervour in the TTD-run SV Gosamrakshana Shala in Tirupati on Thursday Speaking on this occasion the TTD Chairman Sri K.Bapi Raju said according to Hindu mythology Cow is considered as the most sacred living being in the Lord’s creation Worshipping cow is considered to be equivalent to worshipping all the three crore deities of Hinduism “We will be free from all sins if we worship cow It is a great occasion that today hundreds of people converged here to celebrate the Gomahotsavam Director S.V.Go Samarakshna Sala Sri Harinath Reddy and devotees attended the function A flyer for a candidate in a mayoral election north of Tokyo that resembled a poster for an animated movie based on the "Slam Dunk" manga series may have violated copyright law The flyer listing the policies of Mikio Kobayashi who was defeated in Sunday's mayoral election in Kanuma in Tochigi Prefecture including ruling Liberal Democratic Party heavyweight Toshimitsu Motegi Resembling the poster for "The First Slam Dunk," the flyer displayed the phrase "The First Team Up Kanuma" as well as Kobayashi its junior coalition partner the Komeito party and Tochigi Gov which was distributed as a newspaper insert before official campaigning for the election started on June 2 "may have infringed the right to adaptation under the copyright law," said lawyer Hiroyuki Nakajima It reproduced essential features of the movie poster such as the poses of the five figures and the numbers on their uniforms A senior official of Kobayashi's camp said young staff created the flyer after seeing a similar poster displayed on a shopping street in a different prefecture without being aware that it could violate copyright law Japan aims for anime, games sales abroad to hit 20 tril. yen by 2033 Japan football manga "Captain Tsubasa" ends after 43-year run World-first "Dragon Ball" theme park to be built in Saudi Arabia To have the latest news and stories delivered to your inbox Simply enter your email address below and an email will be sent through which to complete your subscription Please check your inbox for a confirmation email Thank you for reaching out to us.We will get back to you as soon as possible Sangareddy: A BRS leader and philanthropist Padamati Anantha Reddy distributed one KG mutton to 400 families on Kanuma festival day at Gunthapally village in Kondapur mandal on Wednesday whose mother worked as a sarpanch in the village to these 400 families before the Sankranthi festival He has been doing various social activities in the village and Kondapur mandal for a decade The villagers thanked him for his gesture on the day of the festival This website is using a security service to protect itself from online attacks The action you just performed triggered the security solution There are several actions that could trigger this block including submitting a certain word or phrase You can email the site owner to let them know you were blocked Please include what you were doing when this page came up and the Cloudflare Ray ID found at the bottom of this page Share your travel photos with us by hashtagging your images with #visitjapanjp These mighty structures are hauled down the streets they face off against each other in a musical showdown From the dazzling waterfront fireworks display to streets lined with food stalls serving tasty treats you can enjoy a truly otherworldly experience and a one-of-a-kind feast for all five senses take the Tohoku Shinkansen bullet train to Utsunomiya Station (about 50 minutes); transfer to the Nikko Line for Kanuma Station (about 15 minutes) https://www.visit-tochigi.com/plan-your-trip/things-to-do/1030/ Browse the JNTO site in one of multiple languages Please view the main text area of the page by skipping the main menu. The page may not be displayed properly if the JavaScript is deactivated on your browser Japanese version Sankranti is a major festival in the Telugu states, celebrated over three days: Bhogi, Makar Sankranti, and Kanuma. It’s a time when people who have moved away for education or work return to their hometowns to celebrate with family. The three days are filled with joy and excitement, and schools and colleges enjoy an extended holiday for the occasion. Both the Andhra Pradesh (AP) and Telangana governments have provided clear details regarding the Sankranti holidays. In AP, the Education Department has confirmed that Sankranti holidays will be observed from January 10 to 19, as per the 2024-25 academic calendar. The holiday dates were adjusted due to recent rains, but despite rumours suggesting a reduction in the holidays, the government has clarified that there is no truth to those claims. SCERT Director Krishna Reddy officially announced the holiday period from January 10 to 19, giving students 10 days off for the festival. Bhogi will be celebrated on January 13, Makar Sankranti on January 14, and Kanuma on January 15. In Telangana, the government has declared Sankranti holidays from January 11 to 17. According to the academic calendar, public holidays are given for Bhogi on January 13 and Makar Sankranti on January 14, while January 15 is an optional holiday. The holidays will begin on January 11, which is the second Saturday of the month, followed by Sunday, and conclude on Friday, January 17. Schools in Telangana will resume on January 18, a Saturday. Additionally, the newly renovated Cherlapalli Railway Terminal in Telangana has been equipped with modern facilities and will operate special trains for Sankranti travelers. Powered by Hocalwire Kanuma Panduga is an essential festival celebrated in the southern states of India Telangana coinciding with the Sankranti festival It falls on the day after Makar Sankranti and is dedicated to honoring cattle This festival marks the sun’s journey toward the northern hemisphere heralding the arrival of the spring season and longer days Welcome to the Current Affairs Section of Adda247 If you are preparing for Government Job Exams then it is very important for you to read the Daily Current Affairs All the important updates based on current affairs are included in this Daily Current Affairs 2024 article Mukkanuma 2025: Kanuma or Mukkanuma is the final day of the Sankranti festival wrapping up the festivities on a happy note Mukkanuma is being observed on 16th January 2025 Mukkanuma 2025: Mukkanuma marks the last day of the vibrant four-day Sankranti festival a day dedicated to thanking hardworking cattle Mukkanuma is all about bonding with loved ones over delicious food and merriment Families and friends reunite to share mouth-watering meals and create lasting memories together As the final day of the Sankranti celebrations Mukkanuma provides a wonderful conclusion to the festivities leaving everyone feeling uplifted and content It's a special day to cherish relationships make new memories and bid farewell to the Sankranti celebrations until next year. This year significance and rituals of this Telugu festival below: Mukkanuma holds great significance as it marks the culmination of the Sankranti festivities This day is dedicated to relaxation and leisure allowing individuals to unwind and recharge after the hectic celebrations sharing traditional delicacies and exchanging gifts The atmosphere is filled with laughter and joy as people bid farewell to the harvest festival and welcome the new season Mukkanuma plays a vital role in Telugu culture This day encourages individuals to strengthen their bonds with loved ones fostering a sense of unity and togetherness Telugu people reaffirm their cultural heritage and traditions strengthening family bonds and leaving everyone feeling joyful and content Mukkanuma is celebrated on the day following Kanuma marking a significant event in the festive calendar this day is observed as Kaanum Pongal in Tamil Nadu highlighting the diverse ways in which festivals are celebrated across different regions Kanuma and Mukkanuma are collectively known as 'Pasuvula Panduga' emphasising the importance of livestock in rural life The main ritual on Mukkanuma is Pasuvula Puja a ceremony dedicated to worshipping cattle In some coastal districts of Andhra Pradesh traditional sports such as Kodi pandaalu (cockfight) and eddula pandaalu (bullock cart races) are organised attracting large crowds and significant betting activity These events add to the festive atmosphere showcasing the rich cultural heritage and traditions of the region ALSO READ: Uzhavar Thirunal 2025: Date, Time, Significance And Rituals Of This Tamil Festival ALSO READ: Mattu Pongal 2025: Date, Time, Significance And Rituals Of The Third Day Of Pongal (Disclaimer: This is based on general public information Jagran English does not confirm its veracity Jagran English brings you all the latest and breaking news from India and around the world Follow english.jagran.com to stay updated with the latest English news Messages And Quotes: The festival is predominantly celebrated in southern India especially in Andhra Pradesh and Telangana It holds profound significance for farmers signalling the commencement of the harvest season and honouring cattle thereby emphasising the vital role of agriculture Happy Kanuma 2025 Messages And Quotes: Kanuma a significant harvest festival is celebrated annually on the fourth day of the three-day Sankranti festival in India It marks the beginning of the sun's journey towards the northern hemisphere signalling longer days and the arrival of spring Kanuma is primarily celebrated in the southern states of India particularly in Andhra Pradesh and Telangana exchange gifts and indulge in traditional delicacies The festival also holds great significance for farmers as it marks the beginning of the harvesting season Kanuma is also associated with the worship of cattle highlighting the importance of livestock in agriculture it's a joyous celebration that brings people together strengthens family bonds and expresses gratitude for the bountiful harvest messages and quotes that you can share with your family and friends May this festival bring prosperity and happiness to your life Celebrate Kanuma with harmony and happiness May your Kanuma be a reflection of the love and unity in your family enriched by traditions and the blessings of this auspicious day 14. Kanuma is a time for gratitude and joy May this festive day strengthen your bonds and bring a bright start to the year May this festival inspire you to embrace gratitude and celebrate life with your loved ones 16. Kanuma reminds us of the beauty of simple joys and deep gratitude Wishing you a festival as bountiful as the harvest Let the joy of this festival uplift your spirits and the blessings of cattle and nature fill your life with abundance 18. Wishing you a Kanuma filled with cherished memories and the blessings of a prosperous year ahead May the traditions of this day strengthen your bonds and the blessings of nature bring abundance to your life may your celebrations be as bright as the sun and your blessings as abundant as the harvest ఈ పవిత్రమైన కనుమ పర్వదినం మీ జీవితంలోని అన్ని చెడులను తొలగించాలని కోరుకుంటూ. కనుమ పండుగ మీ జీవితంలో కొత్త వెలుగులు నింపాలని కోరుకుంటూ.. కనుమ పండుగ మీ జీవితంలో మరుపరాని ఆనందాలను తీసుకురావాలని కోరుకుంటూ హ్యాపీ కనుమ 2025. May the bond between humans and nature grow stronger this festive season and good health on this special occasion of Kanuma Celebrate Kanuma with a grateful heart and joyful spirit Let the festive cheer of Kanuma brighten your days and strengthen your relationships May the festival remind us all of the importance of gratitude and harmony Celebrate Kanuma with gratitude in your heart and joy in your soul 7. Kanuma is a reminder of the harmony between nature and humanity May this festival bring you immense joy and success 8. Kanuma celebrates the essence of nature and gratitude let us be thankful for the blessings of our livestock and the prosperity they bring 10. Kanuma teaches us the value of gratitude and unity May your celebrations be meaningful and joyous this year Kanuma is a time to reflect on our heritage and give thanks May your day be full of meaningful moments and joy let us be grateful for the livestock that enriches our lives and the love that binds us together May this Kanuma bring abundant blessings to your family and moments filled with love and togetherness May the joy of Kanuma fill your heart and home May your festivities be as bright as the love you share with your family and as abundant as the harvest Kanuma teaches us to value life’s simplest blessings and the warm embrace of family and friends Kanuma is more than a festival; it’s a celebration of gratitude and tradition May your day be filled with joyful moments and cherished memories May the festive spirit of Kanuma fill your life with positivity and hope Let this day be a reminder of the strength found in traditions and community 9. Kanuma is a time to reconnect with our roots and express gratitude Wishing you a festival filled with happiness and harmony 10. Sending warm Kanuma wishes to you and your family May your days be filled with love and light ALSO READ: Mukkanuma 2025: Date, Time, Significance And Rituals Of Kanuma Telugu Festival About us | Advertise with us | Contact us Kanuma met the primary endpoint of alanine aminotransferase (ALT) normalisation compared with placebo as well as six secondary endpoints… Data from the Phase 3 ARISE study evaluating the safety and efficacy of Alexion’s Kanuma (sebelipase alfa) in children and adults with lysosomal acid lipase deficiency (LAL-D) have been published Kanuma met the primary endpoint of alanine aminotransferase (ALT) normalisation compared with placebo as well as six secondary endpoints Kanuma is an innovative enzyme replacement for the treatment of patients of all ages with LAL-D and progressive ultra-rare metabolic disease in which infants children and adults suffer multi-organ damage and premature death “Patients with LAL-D often develop cirrhosis and severe dyslipidaemia at an early age historical treatment approaches have not been effective in changing the devastating course of the disease,” said lead study author Barbara K Professor of Paediatrics at the Northwestern University Feinberg School of Medicine and Attending Physician at the Ann & Robert H in this study of children and adults with LAL-D enzyme replacement therapy produced significant reductions in ALT as well as other disease-related lipid and liver abnormalities compared with placebo.” LAL-D is caused by genetic mutations that result in a marked decrease or loss in LAL enzyme activity in the lysosomes across multiple body tissues leading to the chronic build-up of cholesteryl esters and triglycerides in the liver Patients with LAL-D often experience a rapid onset of life-threatening disease manifestations and many patients may be asymptomatic until they experience a severe consequence of the disease “LAL-D is a devastating and ultra-rare disorder in which half of children and adults will progress to fibrosis or liver transplant in three years,” said David Hallal many patients with LAL-D are not accurately diagnosed and those who are face severe consequences… The publication of these pivotal Phase 3 data in the New England Journal of Medicine will raise much-needed awareness of LAL-D in the medical community so patients can receive an accurate and rapid diagnosis.” Alexion announced that the European Commission approved Kanuma for the treatment of patients of all ages with LAL-D the summary of product characteristics (SmPC) for Kanuma in the European Union includes clinical data from a separate study showing a significant benefit in terms of survival (67% or 6 out of 9) in patients with the infant form of LAL-D beyond 12 months compared with 0 out of 21 patients in an untreated historical cohort The study results are published in New England Journal of Medicine All subscriptions include online membership giving you access to the journal and exclusive content By Write for us | Advertise with us European Pharmaceutical Review is published by: Russell Publishing Ltd.Court LodgeHogtrough HillBrasted © Russell Publishing Limited Website development by e-Motive Media Limited Necessary cookies are absolutely essential for the website to function properly This category only includes cookies that ensures basic functionalities and security features of the website These cookies do not store any personal information CookieDescriptioncookielawinfo-checkbox-advertising-targetingThe cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Advertising & Targeting".cookielawinfo-checkbox-analyticsThis cookie is set by GDPR Cookie Consent WordPress Plugin The cookie is used to remember the user consent for the cookies under the category "Analytics".cookielawinfo-checkbox-necessaryThis cookie is set by GDPR Cookie Consent plugin The cookie is used to store the user consent for the cookies in the category "Necessary".cookielawinfo-checkbox-performanceThis cookie is set by GDPR Cookie Consent WordPress Plugin The cookie is used to remember the user consent for the cookies under the category "Performance".PHPSESSIDThis cookie is native to PHP applications The cookie is used to store and identify a users' unique session ID for the purpose of managing user session on the website The cookie is a session cookies and is deleted when all the browser windows are closed.viewed_cookie_policyThe cookie is set by the GDPR Cookie Consent plugin and is used to store whether or not user has consented to the use of cookies It does not store any personal data.zmember_loggedThis session cookie is served by our membership/subscription system and controls whether you are able to see content which is only available to logged in users Kanuma (sebelipase alfa) has been approved for the treatment of patients with a diagnosis of lysosomal acid lipase deficiency (LAL-D)… The US Food and Drug Administration (FDA) has approved Alexion’s Kanuma (sebelipase alfa) for the treatment of patients with a diagnosis of lysosomal acid lipase deficiency (LAL-D) an innovative enzyme replacement therapy (ERT) is the first therapy approved in the US for the treatment of patients with LAL-D Patients with LAL-D (also known as Wolman disease and cholesteryl ester storage disease [CESD]) have no or little LAL enzyme activity This results in a build-up of fats within the cells of various tissues that can lead to liver and cardiovascular disease and other complications Wolman disease often presents during infancy (around 2 to 4 months of age) and is a rapidly progressive disease Patients with Wolman disease rarely survive beyond the first year of life later-onset form of LAL deficiency and presents in early childhood or later Life expectancy of patients with CESD depends on the severity of the disease and associated complications Wolman disease affects one to two infants per million births and CESD affects 25 individuals per million births “I am delighted that patients with LAL-D now have the first approved therapy that treats the underlying cause of the disease,” said Barbara K Professor of Paediatrics at the Northwestern University Feinberg School of Medicine and Attending Physician at the Ann and Robert H LAL-D is nearly always fatal in infants and puts pediatric and adult patients at high risk of vital organ damage and premature mortality 67% of infants who received enzyme replacement therapy survived beyond 12 months of age and children and adults had meaningful improvements in multiple disease-related liver and lipid abnormalities.” The FDA’s Centre for Drug Evaluation and Research (CDER) evaluated the safety and efficacy of Kanuma in an open-label historically controlled trial in nine infants with rapidly progressive Wolman disease and in a double-blind placebo-controlled trial in 66 paediatric and adult patients with CESD In the trial in infants with Wolman disease six of nine infants (67 percent) treated with Kanuma were alive at 12 months of age whereas none of the 21 infants in the historical control group survived there was a statistically significant improvement in LDL-cholesterol levels and other disease-related parameters in those treated with Kanuma versus placebo after 20 weeks of treatment “Patients with LAL-D often suffer for years from a delayed diagnosis only to be further devastated once properly diagnosed because there have been no approved treatments for this disease,” said Brett Billmeyer we are thrilled to welcome the FDA approval of Kanuma providing an approved and effective treatment to patients with LAL-D and their families for the first time and bringing much-needed awareness to this often overlooked and devastating disease.” Kanuma is approved in the United States and European Union , No comments yet Comment *document.getElementById("comment").setAttribute( "id" "afcfdaca9bc4b17e02d25175cac101c2" );document.getElementById("a9d92917df").setAttribute( "id" Metrics details The eggs of the genetically engineered animal contain an enzyme that can treat a rare disease Prices may be subject to local taxes which are calculated during checkout Reprints and permissions Download citation DOI: https://doi.org/10.1038/nature.2015.18985 Anyone you share the following link with will be able to read this content: a shareable link is not currently available for this article Sign up for the Nature Briefing: Translational Research newsletter — top stories in biotechnology Kanuma™ (sebelipase alfa) is an enzyme replacement therapy (ERT) for the treatment of patients of all ages with lysosomal acid lipase deficiency (LAL-D) Kanuma™ (sebelipase alfa) is an enzyme replacement therapy (ERT) for the treatment of patients of all ages with lysosomal acid lipase deficiency (LAL-D) The drug was discovered and developed by Synageva BioPharma which was acquired by Alexion Pharmaceuticals in June 2015 Alexion received marketing authorisation approval for Kanuma from the European Commission (EC) and the US Food and Drug Administration (FDA) in September 2015 and December 2015 respectively making it the first FDA-approved therapy for that indication The new drug application (NDA) for Kanuma was submitted to the Japan’s Ministry of Health Lysosomal acid lipase deficiency is a fatal inherited lysosomal storage disease caused by harmful mutations in the LIPA gene The disease results in accumulation of cholesteryl esters and triglycerides in the liver It affects patients of all ages from infancy through adulthood approximately 50% of children and adults with LAL-D progressed to fibrosis cirrhosis or liver transplant in three years The median age of onset of LAL-D is 5.8 years It is a rare disease that affects one to two infants out of one million births which addresses the underlying cause of LAL-D and also reduces substrate accumulation in the lysosomes of cells throughout the body The drug is available in 20mg/10ml dose for intravenous administration The US FDA approval for Kanuma was based on the results obtained from two phase III clinical trials The first clinical study was an open-label which was conducted on nine infants with LAL deficiency The patients received Kanuma at 0.35mg/kg once weekly for the first two weeks and then 1mg/kg once weekly They were compared with an untreated historical cohort of 21 patients The study results showed that of the nine Kanuma-treated infants while 21 patients in the historical cohort arm died by eight months of age The median age of six surviving Kanuma-treated patients was 18.1 months The study also showed that after the initiation of treatment with Kanuma 1mg/kg once weekly weight-for-age z-scores improved in three of five surviving patients The dose escalation to 3mg/kg once weekly resulted in all six surviving patients improving in weight-for-age z-scores The most commonly reported adverse events in clinical trials in the infants treated with Kanuma were diarrhoea The second clinical trial evaluated the safety and efficacy of Kanuma The study enrolled 66 paediatric and adult patients with LAL-D The patients were randomised to receive Kanuma at a dosage of 1mg/kg or placebo once every other week for 20 weeks in the double-blind period The study results demonstrated that a statistically significant improvement in percent change from baseline in LDL-c was observed in the Kanuma-treated group compared to the placebo group after a 20-week double-blind period of the trial The most commonly reported adverse events in clinical trials in the Kanuma-treated paediatric and adult patients included headache Give your business an edge with our leading industry insights View all newsletters from across the GlobalData Media network Newsletters and Deep Dive digital magazine Alexion has gained FDA approval for Kanuma its treatment for the rare disease lysosomal acid lipase (LAL) deficiency Alexion says Kanuma (sebelipase alfa) could eventually bring in more than $1 billion in annual sales helping it to sustain its growth in the rare disease field The drug follows close behind another new Alexion rare disease drug which gained FDA approval in October for paediatric-onset hypophosphatasia (HPP) which until now has rested on Soliris (eculizumab) a treatment for two rare and life-threatening diseases Combined sales for its use in Paroxysmal nocturnal haemoglobinuria and atypical haemolytic uremic syndrome helped it generate sales of $2.23 billion last year but the company needs to add new products to its portfolio to maintain its rapid growth inherited condition caused by the absence of the LAL enzyme which breaks down certain lipids; without it these fats accumulate in organs leading to fatty liver disease The US regulator granted Kanuma breakthrough therapy designation in 2013 where the condition is known as Wolman disease The condition presents around 2-4 months of age as a rapidly progressive disease and patients rarely survive beyond the first year of life The FDA's CDER evaluated the safety and efficacy of Kanuma in an open-label placebo-controlled trial in 66 pediatric and in CESD six of nine infants (67%) treated with Kanuma were alive at 12 months of age The FDA had been due to rule on the drug in September but asked Alexion for more information on the chemistry One of the reasons for this was the novel manufacturing process used to produce the enzyme – genetically engineered chickens These 'GE' chickens carry the rDNA construct which produces the rhLAL protein in their egg whites The FDA wanted longer to probe the process and used both its human and veterinary health divisions Alexion says the GE chickens are used only for producing the drug substance and neither the chicken nor the eggs are allowed in to the food supply The US price of Kanuma will be eagerly awaited by analysts and other industry watchers the price of rare disease drugs have been attracting lots of criticism in 2015 Alexion's shares fell in October when it announced an annual average price of $285,000 for Strensiq well below the $400,000 analysts had been expecting Despite this lower-than-expected US price, Strensiq has already been rejected in draft guidance from England's cost effectiveness watchdog NICE The drug is priced at £366,900 ($552,000) in England and Alexion will now have to enter pricing negotiations in order to change NICE's mind While Alexion has yet to announce Kanuma's US price analysts at Barclays recently forecast an annual cost of $375,000 Alexion could set Kanuma at a similar level Alexion expands its reach in ultra-rare disease The EU has announced a €500m scheme to convince scientific talent from around the world to relocate to Europe's "free and open" research environment The first biosimilars of AstraZeneca/Alexion's blockbuster complement C5 inhibitor Soliris have been launched in the US at a discount to the brand Understanding how to harness real-world data and digital insights is essential The importance of biosimilars only continues to grow driven by the potential savings they are able to deliver to healthcare systems Jonah Comstock welcomed back Alice Valder Curran to the podcast to pick apart Trump's Executive Order order piece by piece Developments in the oncology space in 2024 brought hope to both industry and patients The ESG in Life Sciences Summit West is coming to San Francisco The ISPE Singapore Affiliate Conference & Exhibition is one of the largest and most respected events for bio & pharmaceutical manufacturing professionals opinions and features on pharma and healthcare sent straight to your inbox on Tuesday 28th January @ 10 am ET - 3 pm GMT - 4 pm CET entitled ‘Health Trends 2025: Reimagining What's Possibl Sign up for email newsletters and Deep Dive Alexion has announced that the CHMP of the EMA has adopted positive opinions recommending marketing authorisation of Strensiq and Kanuma… Alexion has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted positive opinions recommending marketing authorisation of Strensiq and Kanuma The proposed indication for Strensiq is for long-term enzyme replacement therapy in patients with paediatric-onset hypophosphatasia (HPP) to treat the bone manifestations of the disease The proposed indication for Kanuma is for long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency (LAL-d) Based on the CHMP’s positive recommendations final decisions from the European Commission are expected in the third quarter of 2015 there are no therapies approved for the treatment of HPP or LAL-d “The CHMP positive opinions for Strensiq and Kanuma are significant milestones in bringing these therapies to infants and adults suffering from HPP and LAL-d in Europe,” said David Hallal “Both Strensiq and Kanuma are highly innovative enzyme replacement therapies that will be the first treatments available for patients with HPP and LAL-d two life-threatening and ultra-rare metabolic disorders.” ultra-rare metabolic disease in which patients experience devastating effects on multiple systems of the body leading to debilitating or life-threatening complications It is characterised by defective bone mineralisation that can lead to deformity of bones and other skeletal abnormalities as well as systemic complications such as profound muscle weakness pain and respiratory failure leading to premature death in infants ultra-rare metabolic disease in which patients ranging from infants to adults experience chronic lipid accumulation causing multi-systemic organ damage and premature death It is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues leading to the chronic build-up of fatty material in the liver , Alexion Pharmaceuticals plans to acquire Synageva BioPharma for $8.4 billion in a deal that will expand the buyer’s portfolio of metabolic treatments with what it boasted will be “the most robust rare disease pipeline in biotech.” That pipeline will include Synageva’s Kanuma™ (sebelipase alfa) an enzyme replacement therapy for lysosomal acid lipase (LAL) deficiency for which regulatory decisions are expected later this year from the FDA and the European Commission A recombinant form of the human LAL enzyme Kanuma has been granted the FDA’s Breakthrough Therapy Designation for LAL deficiency in infants and is under priority review by the agency Kanuma has been given accelerated assessment of its Marketing Authorization Application by the European Medicines Agency (EMA) Labour and Welfare (MHLW) have granted Kanuma their orphan drug designations “As Kanuma moves closer toward patients who suffer from LAL deficiency and the other pipeline programs continue to progress I am confident that this transaction will help continue to improve the lives of patients with LAL deficiency and other devastating “Alexion is uniquely suited to advance Synageva’s mission to deliver life-saving therapies to patients whose diseases were once considered too rare for developing treatments.” Kanuma is one of two Synageva-developed treatments expected that Alexion expects to launch this year a first-in-class enzyme replacement therapy intended to address the underlying cause of hypophosphatasia (HPP) Strensiq is designed to restore the genetically defective metabolic process thus preventing or reversing potentially life-threatening complications of lifelong dysregulated mineral metabolism The FDA has also granted Breakthrough Therapy designation for Strensiq and accepted the company’s Biologics License Application (BLA) for Priority Review Alexion has an MAA pending with the EMA for Strensiq and has submitted an NDA for Strensiq to MHLW Alexion expects Strensiq and Kanuma to join a portfolio of rare-disease treatments anchored by Soliris® (eculizumab) a first-in-class terminal complement inhibitor and elsewhere as the first and only treatment for patients with both paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis and the first and only treatment for patients with atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy or TMA Soliris generated more than $2 billion in revenues last year The Synageva acquisition will expand Alexion’s pipeline to eight product candidates in clinical trials for eleven indications an enzyme replacement therapy in an ongoing Phase I/II trial for mucopolysaccharidosis IIIB (MPS IIIB) SBC-103 won the FDA’s Fast Track in January The deal will also give Alexion more than 30 preclinical programs including 12 from Synageva’s drug discovery platform Alexion expects at least four preclinical candidates to begin clinical trials by the end of 2016 Synageva also brings to Alexion its expression platform manufacturing technology—a system of vectors designed for producing proteins with human-like glycosylation patterns—and three upstream manufacturing facilities Alexion plans to acquire all outstanding shares of Synageva common stock through an exchange offer Alexion will exchange each Synageva share for $115 cash and 0.6581 shares of Alexion stock The stock portion of the deal is expected to be tax-free to Synageva stockholders The deal is subject to customary closing conditions the tender of a majority of the outstanding shares of Synageva common stock and receipt of required regulatory approval The transaction is expected to close mid-2015 Alexion can carry out a one-step merger through a vote of Synageva stockholders including affiliates of Baker Brothers Investments have agreed to exchange about 33.5% of Synageva’s outstanding shares with Alexion which has received $3.5 billion in financing from Bank of America Merrill Lynch and J.P Copyright © 2025 Sage Publications or its affiliates including those for text and data mining and training of large language models The two events are significant not just because of their impact on health is the only treatment for lysosomal acid lipase deficiency and ZMapp remains the only effective treatment for Ebola (and it is still progressing through clinical trials) The even bigger impact is that these two drugs might represent the resurgence of biopharming — the creation of pharmaceuticals grown through plants or farm animals Biopharming started in the late 1980s with a lot of promise scientists reported that monoclonal antibodies could be made by tobacco plants That opened the door to a number of drugs to be made in a similar fashion Scientists saw this as a potential way to treat diseases more cheaply as these methods are cheaper than using traditional chemical or bio-production methods rice or any number of crops were seen as close to reality Hundreds of biotech companies applied to the U.S Department of Agriculture’s Animal and Plant Health Inspection Service (APHIS) —  the bureau charged with approving and monitoring field trials and development of these new drugs — for permission to make ‘farmaceuticals.’ one of the more even-handed organizations looking at genetic modification even if USDA develops stringent regulations for pharma crops mistake in such large-scale agricultural productions is high As the USDA tightened rules for approval of experimental field trials investors began to scatter like so much stray corn seed Monsanto dropped its research programs in plant derived biotechnology In addition to regulatory and public relations problems “pharming” was also running into some technical walls Plants didn’t always express enough of the desired protein and purification of the expressed protein was proving to be a major issue plant-expressed (or animal-expressed) proteins often could trigger an immune reaction in humans which will drastically reduce the risks of an immune reaction to the drug which were the bone of contention after the ProdiGene incident have largely been replaced by sealed bioreactors Both solutions have avoided the problem of “contamination” of food with plants (or animals) bred for drug production a major regulatory achievement according to both the USDA and FDA And, as ZMapp and Kanuma have shown, some movement in pharming is happening. A number of other plants and animals have been approved for drug production, and the list of permits issued by APHIS has started to surge Biopharming offers tremendous flexibility and economy when adjustments in production are necessary Another advantage is that it offers great potential for cost cutting: The energy for product synthesis comes from light (sun or artificial) and the primary raw materials are water and carbon dioxide Andrew Porterfield is a writer, editor and communications consultant for academic institutions, companies and non-profits in the life sciences. He is based in Camarillo, California. Follow @AMPorterfield on Twitter Infants with a rare and fatal congenital disorder called Wolman disease in England and Wales will soon have access to a new drug via the NHS that can dramatically extend their lives The National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending Kanuma (sebelipase alfa) – sold by AstraZeneca’s Alexion unit – after the company reached a commercial deal with NHS England There are currently less than a dozen children in the UK with Wolman disease an infant-onset form of lysosomal acid lipase deficiency (LAL-D) which is generally fatal within the first year of life Alexion originally sought approval for all forms of LAL-D, which affects around 25 people, but that was turned down in 2017 NICE agreed to start new guidance development under the highly specialised technology (HST) pathway The missing LAL enzyme causes a rapid build-up of fat in the liver jaundice and anaemia and eventually multiple organ failure and death Kanuma, which costs £300,000 ($378,000) a year at list prices and can be given as a weekly infusion at home, will now start to be made available on the NHS with funding under the £340 million Innovative Medicines Fund (IMF) set up last year to fast-track NHS access to promising new drugs with limited data backing them up Kanuma is one of the first medicines to be made available under the scheme Children will have to take it for life or until they are able to receive a stem-cell transplant a specialist in childhood inherited metabolic diseases at the Royal Manchester Children’s Hospital and Saint Mary’s Hospital said the deal “marks a major milestone in treatment for infants born with Wolman disease and signifies a substantial step forward in our dedication to practical advancements in rare disease medicine and improved patient outcomes Kanuma will be delivered by specialist services at London’s Great Ormond Street Hospital Birmingham Women’s and Children’s Hospital was diagnosed with Wolman disease aged three months and began the treatment in January 2016 as part of a clinical trial at RMCH His father Jabran said he would not be alive today without treatment but thanks to the drug he is “able to live a normal life – going to school full-time started treatment with sebelipase alfa when he was three days old After getting the drug for more than two years he was eventually able to receive a stem-cell transplant and no longer requires treatment “Today is a milestone moment for infants born with Wolman disease and their families,” commented Sean Richardson “The recommendation is the result of continued constructive collaboration between Alexion to ensure babies born with this life-threatening disease have a treatment available to them.” Photo by Alexander Grey on Unsplash Rare disease research is vital to improve the lives of hundreds of millions of people worldwide The US Food and Drug Administration (FDA) today approved sebelipase alfa (Kanuma the first treatment for a rare genetic condition called lysosomal acid lipase (LAL) deficiency The new drug is a recombinant form of the human LAL enzyme produced in the egg whites of genetically engineered chickens This recombinant human LAL makes up for the missing or inactive LAL in humans with the deficiency LAL deficiency causes cholesterol esters and triglycerides to build up in vital organs such as the liver which can lead to liver and cardiovascular disease and other complications A rapidly progressive form of the condition called Wolman disease often presents in infants at 2 to 4 months of age These infants rarely survive beyond a year Cholesteryl ester storage disease (CESD) is a milder version of LAL deficiency that presents later in childhood and beyond life expectancy depends on disease severity The genetically modified chickens that contribute the crucial replacement enzyme are not allowed in the nation's food supply "LAL deficiency is a rare inherited genetic disorder that can lead to serious and life-threatening organ damage especially when onset begins in infancy," said Janet Woodcock director of the FDA's Center for Drug Evaluation and Research (CDER) these patients for the first time ever have access to a treatment that may improve their lives and chances of survival." Two separate FDA offices signed off on the new drug The agency's Center for Veterinary Medicine approved the recombinant DNA construct in the genetically engineered chickens that codes for the replacement enzyme CDER determined that sebelipase alfa was safe and effective on the basis of two trials historically controlled trial in nine infants with Wolman disease who were treated with the drug Six of the nine infants were alive at 12 months of age none of the 21 infants in the historical control group lived that long involved 66 pediatric and adult patients with CESD patients treated with sebelipase alfa posted statistically significant improvements in low-density lipoprotein cholesterol levels and other disease-related metrics compared with patients receiving the placebo the Center for Veterinary Medicine assessed both the safety of the recombinant DNA construct in chickens and its stability in the animal's genome over several generations The Center for Veterinary Medicine also determined that approving the genetically engineered chickens for medicinal purposes "does not cause any significant impact on the environment because the chickens are raised in highly secure indoor facilities," the FDA said The most common adverse effects observed in patients treated with sebelipase alfa are diarrhea More information about today's announcement is available on the FDA website Medscape Medical News © WebMD, LLC Heartwire © WebMD, LLC WebMD Health News © WebMD, LLC Reuters Health Information © Send comments and news tips to news@medscape.net Robert Lowes is a journalist for Medscape Medical News A former senior editor at Medical Economics magazine and contributor to numerous healthcare publications Robert has covered medicine from almost every conceivable angle — public policy His articles have won major awards such as first place in the annual journalism competition of the National Institute for Health Care Management and several have been republished in books He can be contacted at rlowes@medscape.net Click the topic below to receive emails when new articles are available You will receive email when new content is published The majority of food consumed in The Gambia is imported but a growing number of young entrepreneurs are starting food production companies encouraged by the availability of UN-supported training Alhadgie Faal has built a successful small business where he grows fruit and vegetables to sell to restaurants and hotels He started his company after receiving training from a UN Capital Development Fund (UNCDF) programme[DD1]  Without agriculture we can’t feed the nation and I had a dream to supply The Gambia with healthy produce my stepfather told me that free UN training in agriculture because we are a farming family with little money and I would not have been able to afford fees and transport But all of these expenses were included in the offer I got the idea to start my own fruit and vegetable business and she allowed me and my brother to use it so my brother and I cleared it to create a garden we protect the crops with netting to protect them from rodents and we cover them with plastic for three to four days to generate heat We have been successful in selling our fruit in the Banjul region Many people like our produce because it’s very tasty and all of our fruit and vegetables are organic because chemicals are not good for human health or the environment Starting an agriculture business in The Gambia is very hard because it’s hard to get access to grants and loans My advice for anyone who wants to be an entrepreneur is to focus on agriculture so that we can avoid importing from our neighbouring countries I would like Gambians to eat Gambian strawberries People have the misconception that we don’t grow the fruit here Mr. Faal’s training, a three month course on horticulture vegetable production at Gambia Horticultural Enterprise (GHE), was provided as part of the Jobs, Skills and Finance (JSF) programme for Women and Youth in The Gambia, the flagship programme of the UN Capital Development Fund (UNCDF), in collaboration with International Trade Center (ITC) and EUR 15 million of funding from the European Development Fund JSF addresses persistent challenges in The Gambia which include lack of job opportunities for youth and women, low levels of financial inclusion and climate change adaptation and mitigation. Climate adaptation activities are delivered using the Local Climate Adaptive Living Facility The objective of JSF is to provide support to local training providers and accessibility of vocational training schemes offered to youth and women which are linked to the Cash for Work (CfW) opportunities Local training institutions are contracted to provide training in different skill areas highly demanded by the labour market The programme supports Target 8.3 of Sustainable Development Goal 8 which calls for the promotion of development-oriented policies that support productive activities and encourage the formalization and growth of micro including through access to financial services Find out more about the work of the UN in The Gambia here she reflects on her experience over the last five years which have seen the country emerge from a long period of authoritarian rule A company that began in a University of Georgia laboratory commanded one of the biggest price premiums in history in a multi-billion dollar takeover when the company sold this summer It's another winner in the fast-emerging orphan drug market Connecticut-based Alexion Pharmaceuticals announced in May it would pay $8.4 billion for Synageva BioPharma originally called Avigenics when UGA geneticist Bob Ivarie and businessman George Murphy founded the company nearly 20 years ago The price Alexion agreed to pay was more than double Synageva's market value - all on the promise of a single drug Synageva was on the verge of taking to market could have sales of as much as $1 billion a year in the so-called orphan drug market Alexion hasn't yet set a price for the new drug the director of UGA's technology commercialization office said at a recent meeting of the UGA Research Foundation board of directors UGARF is a nonprofit corporation controlled by the university that manages and promotes research and commercializing research But analysts said in May the price of Kanuma could be as much as $350,000 a year or even more - the literal cost of life for infants born with the most severe form of a very rare and often fatal genetic condition called lysosomal acid lipase deficiency Kanuma is already approved for use in Europe The orphan drug market wasn't on Bob Ivarie's mind when he founded the company He was focused more on "how" than "what" back then - how to genetically engineer chicken hens into little pharmaceutical factories bioreactors that could be programmed to make protein-based medicines Ivarie wasn't the first scientist to think about the possibility of altering chickens genetically to produce protein-based medicines in chicken eggs which are almost entirely protein and water "The idea has been around for a while," said Ivarie now retired from UGA and no longer active in the company he founded Ivarie wanted to take a shot at figuring out how it could really be done He took the idea to administrators at the UGA Research Foundation They thought it a great idea and put him in touch with a California investor named George Murphy The foundation also helped Avigenics along in other ways such as obtaining patent protection for the techniques Ivarie and a growing team of scientists developed on-campus housing for the budding company in UGA's Georgia Biobusiness Center A organization funded by both public and private dollars called the Georgia Research Alliance also helped by providing lab equipment so the young company could use investor dollars to hire people to help in the growing research effort The GRA contributed $5 million toward a UGA's "Biobusiness Center," a campus building housing start-up companies where Avigenics was for several years a tenant The Avigenics team's first step was to show it could be done it also thought of how its techniques might prove commercially viable One early idea was to modify its white Leghorns to produce large amounts of interferons signaling proteins that can boost the body's natural immune systems to fight a wide variety of ills But a new line of thought emerged in 2008 with new management and investors after the company changed its name from Avigenics to Synageva and as has often been the case with Georgia-founded biotech companies That new thinking was to use the transgenic chicken and egg techniques to enter the increasingly lucrative orphan drug market alongside companies such as Alexion which has the distinction of producing what at one time was the world's most expensive drug according to press accounts of the Synageva sale earlier this year often devastating medical conditions affecting small numbers of people estimates are around 3,000 to 4,000 worldwide drug companies spent relatively little money researching possible drugs for such orphan diseases But a new business model has emerged in recent years Orphan drugs have become a fast-growing part of the pharmaceutical market thanks in part to a business strategy of sky-high pricing regulatory policies that speed up approval and government incentives such as federal and state funding for the underlying research the only treatment for a couple of rare conditions in which the body destroys its own red blood cells and one that is literally a lifesaver for those who need it At a cost of about $700,000 a year for the 180 Canadian patients who depend on it it's one of the world's most expensive drugs according to Canada's National Post; the price in the United States is somewhat lower It has also gained approval for another orphan drug in addition to Soliris and Kanuma The UGA Research Foundation at one time had a financial stake in Avigenics and Synageva but won't profit from the landmark takeover this summer UGA often takes a small part of ownership in companies started by university researchers Eberhart couldn't say this week what UGA's stake was in the company though the percentage would be diluted over time as investors poured tens of millions of dollars into the company UGARF sold 1,500 shares of its Synageva common stock in June 2012 for $56,286 and its remaining 2,223 shares in December 2012 for $122,655.39 - a good price at the time absent a crystal ball that would have foretold the blockbuster Alexion deal that would come three years later In announcing the Synageva purchase this spring Alexion estimated the worth of its offer at $230 a share - for UGA's 3,723 shares UGARF still stands to get licensing income from Alexion a small percentage of the revenue the company gets from selling Kanuma and possible future drugs the Synageva chickens may produce UGARF holds the patents for a number of discoveries Ivarie and his company made UGA researchers get a portion of any patent income along with UGARF "It has the potential to be a very significant royalty stream," Eberhart said Patents UGARF licensed exclusively to Synageva expire between 2017 and 2024 according to a filing the company made with the federal Securities and Exchange Commission Avigenics/Synageva is a significant success story for UGA and its research foundation said UGA Vice President for Research David Lee "(Kanuma) will make a significant difference in the lives of people who have very little hope," Lee said "We can all take a lot of pride in the fact that the university made a real difference in their lives." Ivarie judges Avigenics a success story in a couple of other ways It created a lot of jobs and it's an economic development success story in that a viable company grew out of university research And it showed that a successful and important biotech company can grow in Georgia Follow education reporter Lee Shearer at www.facebook.com/LeeShearerABH or https://twitter.com/LeeShearer Alexion has hit out at NICE's rejection of ultra rare disease treatment Kanuma - the second block on its drugs in just eight days Last week NICE rejected Strensiq also for an ultra-rare condition mainly affecting children In both cases NICE said it couldn't recommend the drugs because of doubts about their benefits for patients Strensiq has a list price of £366,000 ($457,000) per patient per year but Kanuma's is even higher - NICE estimated it would cost £491,992 per patient per year.  This makes Kanuma the most expensive drug NICE has ever reviewed eclipsing Alexion's former holder of the title 'most expensive drug in the world' While Alexion has offered a confidential discount on both Strensiq and Kanuma [caption id="attachment_24311" align="alignnone" width="255"] Carole Longson[/caption] NICE health technology evaluation centre director said: “Throughout this extremely complicated evaluation we have given the company many opportunities to improve the terms under which sebelipase alfa could be offered to patients even their best offer to date falls far short given the considerably uncertainties about its longer term benefits and its very high cost.” Kanuma (sebelipase alfa) is a treatment for lysosomal acid lipase deficiency (LAL-D) LAL deficiency which affects around 25 people in England but can be a devastating condition particularly when it progresses rapidly in babies less than six months old Alexion proposed that Kanuma would have been made available with an annual per-patient cost cap The committee heard from the patient experts that Kanuma offered a potential lifeline for babies with this form of the condition who have no other treatments available to them the committee concluded that the benefits of treatment were uncertain and the cost was too high for these benefits Alexion has criticised NICE’s the body’s Highly Specialised Technology saying its process is “inconsistent” and “ineffective” “NICE’s decision to not recommend Kanuma for the treatment of patients with LAL-D is an injustice to patients in England and based on arbitrary cost-effectiveness measures," a spokeman said could result in devastating consequences for patients in England with LAL-D who may face significant suffering multi-organ damage and potential premature death." The company reiterated that the decision reinforced how England and Wales were falling behind other countries in funding medicines for patients suffering from rare and ultra-rare diseases with limited treatment options There is some hope that NICE and Alexion could come to an agreement on Strensiq and Kanuma In 2014 NICE recommended Alexion’s Soliris (eculizumab) for the rare kidney disease atypical haemolytic syndrome (aHUS) albeit after a lengthy delay while the government reorganised assessments of ultra-rare disease drugs Formerly described as the world’s most expensive drug NICE estimated Soliris' cost at around £340,000 per patient annually at full price  - far lower than Kanuma's £491,992 NICE has a list of further rare disease drugs it intends to evaluate This includes the drug now believed to be the world's most expensive: Chiesi's gene treatment Glybera which costs around 900,000 euros or approximately £766,500 Stand-offs between NICE and companies with rare disease drugs could become even more common, as the cost effectiveness watchdog is proposing a new threshold for so-called highly specialised technologies. NICE and payer NHS England have proposed a new QALY threshold allowing automatic funding for very rare condition treatments up to £100,000 per QALY with products above this cut-off point being considered through NHS England’s prioritisation framework AD Leave a rating/comment#PrintsBack to ArticlesSHARE A company that began in a Uni­versity of Georgia laboratory commanded one of the biggest price premiums in history in a multibillion-dollar takeover when the company sold this summer Connecticut-based Alexion Phar­ma­ceuticals announced in May it would pay $8.4 billion for Syna­ge­va BioPharma originally called Avi­genics when UGA geneticist Bob Ivarie and businessman George Murphy founded the company nearly 20 years ago But analysts said in May that the price of Kanuma could be $350,000 a year or more - the literal cost of life for infants born with the most severe form of a very rare and often fatal genetic condition called lysosomal acid lipase deficiency The orphan drug market wasn't on Ivarie's mind when he founded the company in 1996 Ivarie wasn't the first scientist to think about altering chickens genetically to produce protein-based medicines in chicken eggs He wanted to take a shot at figuring out how it could really be done and took the idea to administrators at the UGA Research Foundation They put him in touch with a California investor named George Murphy The foundation helped Avigenics along in other ways A organization funded by public and private dollars called the Georgia Research Alliance also helped The alliance contributed $5 million toward UGA's Bio­business Center a campus building housing start-up companies where Avi­genics was a tenant for several years signaling proteins that can boost the body's natural immune systems to fight a variety of ills according to news accounts of the Synageva sale earlier this year drug companies spent relatively little money researching drugs for such orphan diseases But orphan drugs now have become a fast-growing part of the pharmaceutical market regulatory policies that speed approval and government incentives for the underlying research and one that is a lifesaver for those who need it according to Canada's National Post; the price in the U.S It has gained approval for another orphan drug in addition to Soliris and Kanuma The UGA Research Foundation at one time had a financial stake in Avi­genics and Synageva It stands to get licensing income from Alexion - a small percentage of the revenue the company gets from selling Kanuma and possible future drugs the Synageva chickens produce please disable the ad blocking feature and reload the page This website uses cookies to collect information about your visit for purposes such as showing you personalized ads and content By clicking “Accept all,” you will allow the use of these cookies Users accessing this site from EEA countries and UK are unable to view this site without your consent By Wakako Takeuchi / Yomiuri Shimbun Staff Writer Kanuma kumiko is a handicraft associated with the city of Kanuma in Tochigi Prefecture in which wooden strips are latticed in geometric patterns are a long-standing fixture in Japanese-style rooms and are often used for shoin-style paper sliding doors and transoms the craft has begun to be utilized for a wider range of purposes including wall decorations and smaller items such as coasters The technique is said to have originated among woodworkers who gathered from across the country to build Nikko Toshogu Shrine during the Edo period (1603-1867) Kanuma kumiko eschews nails and metal fittings Experienced craftsmen carve notches into finely cut wooden pieces and fit them together with flawless precision The Tochigi prefectural government has designated Kanuma kumiko as a traditional handicraft and the Kanuma city government has created a related “Kanuma” brand Yoshihara Mokugei President Koji Yoshihara who founded the city-based company at age 31 works with his three sons to produce traditional kumiko handicrafts All four have been designated as traditional craftsmen by the prefecture Kumiko craftspeople primarily work with Japanese cedar and cypress trees grown in Kanuma in addition to cypress trees from the Kiso region Yoshihara has developed other patterns through a process of trial and error including “kujaku” (peafowl) and “Fujisan” (Mt There are “countless possibilities” for kumiko designs Yoshihara professes to a fascination for the deep geometrical beauty associated with the craft Although he has been involved in its production for more than half a century I feel always moved when I put in the last part,” he said traditional handicrafts have increasingly been seen in a positive light Kanuma kumiko now features in a corner of the Kanuma city hall that was completed in April the long-term outlook for the handicraft is uncertain due to a decrease in orders related to a decline in Japanese-style buildings a commercial and industrial association for the city’s woodworkers says the shipment value of fittings has been in decline since its peak in the early 1990s Fitting makers also have been troubled by imitations and non-official products the association is preparing to register local products under the Japan Patent Office’s “Regional Collective Trademark System” to protect the brand producers will be able to claim damages when imitations or copies are put on sale it is possible that kumiko products may become pricier Instability in the foreign-produced wood market has resulted in increased use of domestic wood This is expected to raise prices for thick trees Such timber is necessary to produce kumiko were to become out of reach of ordinary people,” Yoshihara said “I’d like to produce a wider variety of products Our weekly ePaper presents the most noteworthy recent topics in an exciting © 2025 The Japan News - by The Yomiuri Shimbun Okinawa — A small wooden tablet that belonged to a Japanese soldier killed during the Battle of Iwo Jima was returned to his family in December thanks to the efforts of a Texas-based researcher a shipping tag that bears a handwritten list of items that belonged to her uncle during a ceremony at Kanuma City Hall on Dec she told Stars and Stripes by phone Monday Murota was just 24 when he died defending the Japanese island stronghold during the last year of World War II The 7-inch by 3-inch tablet was retrieved from the island by U.S Navy sailor Raymond Sexton and later donated to the Minnesota Military Museum It was returned at the suggestion of war researcher and Iwo Jima Association of America volunteer “I had seen on TV that those kinds of things happen but I never thought that it would happen to me.” The Battle of Iwo Jima began with a Marine amphibious assault on Feb following months of aerial and naval bombardment Seventy-thousand Marines took part in the 36-day battle with more than 6,800 killed and 19,000 wounded The well-preserved piece of wood is addressed to Apprentice Officer Morihiro Murota from his father medicine and pencils that were included in the original package Murota was assigned to the 311th Independent Infantry Battalion at the time of his death then a 21-year-old Seabee with the 106th Construction Battalion was building integral infrastructure on the island when he collected the tag as a wartime souvenir Sexton’s son donated the tablet to the museum after his father was hospitalized due to “old age,” the statement said author of “A Tomb Called Iwo Jima: Firsthand Accounts from Japanese Survivors,” suggested returning the tablet to Murota’s family after seeing it in a Facebook post by museum curator Doug Thompson He sent a letter to Kanuma city officials in August aided by retired Kyoto-based real estate executive Nobuhiro Nakamura It took them only a few weeks to track down Kawarai Kawarai officially received the tablet and several vials of black volcanic sand from Iwo Jima’s beach from Kanuma Mayor Shin Sato on Dec Seeing the piece of wood was heartbreaking “I knew about the war but after having the wooden plate in my hands I could have a real feeling,” she said She was devastated by her brother’s death and traveled to Iwo Jima about 20 years ago to try She never learned about the wooden tablet’s existence due to dementia Kawarai placed the relic next to the memorial tablet on her mother's altar at home She plans to show it to another of Murota’s surviving siblings Daniel BuenoThe first transgenic chicken approved by the Food and Drug Administration (FDA) – the agency that regulates the food and drug industry in the United States – will not be used for human consumption but rather as a biofactory for producing the enzyme sebelipase alfa in its eggs in the form of an injectable drug made by the pharmaceutical company Alexion is the first treatment for lysosomal acid lipase (LAL) deficiency a rare genetic disorder also known as Wolman disease can kill babies in their first six months of life The disorder leads to the build-up of fat in the liver Without lysosomal acid lipase – or if it fails to work properly – a person’s ability to break down esterified cholesterol and triglycerides is seriously diminished Wolman disease strikes once every one million births the drug is purified from the egg whites of the transgenic hens The FDA verified that the enzyme does not affect the health of the animals The company will raise the hens in a secure environment and they will not enter the food supply The FDA designated Kanuma as an orphan drug making it eligible for such financial incentives as tax credits © Revista Pesquisa FAPESP - All rights reserved. by Arielle Duhaime-Ross Source FDA | Via Nature FacebookThreadsJohnath / FlickrYesterday, the US government approved a genetically modified chicken The drug in question is called Kanuma, reports Nature. It’s the first treatment approved for people with “lysosomal acid lipase deficiency” — a rare genetic condition that prevents the body from breaking down fatty molecules inside cells. Unlike the FDA’s recent approval of genetically modified salmon these chickens and their eggs aren’t approved for human consumption But yesterday’s approval doesn’t rely on milk production researchers have to purify it from the whites of the chicken’s eggs Kanuma works by replacing a malfunctioning enzyme in people with lysosomal acid lipase deficiency As part of the approval process for the drug the government officials looked at whether the alterations made to the chicken’s genetic material would cause it harm They also examined whether these changes are stable enough to be passed on to future generations Correction: A previous version of this article incorrectly stated that Bernadette Dunham is the director of the CDC’s Center for Veterinary Medicine. In fact, she is the director of the FDA’s Center for Veterinary Medicine. We regret the error. A weekly newsletter by David Pierce designed to tell you everything you need to download, watch, read, listen to, and explore that fits in The Verge’s universe. 16 Jan 2016: Every day is a festival day in Tirumala and if three festivals coincides on a single day Saturday witnessed similar religious fervour in the hill town as three festivals including Kaka Bali Goda Parinayam and Paruveta Utsavam were performed in Tirumala temple on the auspicious Kanuma day GODA PARINAYAM: Later in the day at around 10am the chief pontiff of Pedda Jiyangar Mutt handed over special Andal Sri Goda Devi garlands brought from Sri Govinda Raja Swamy temple These sacred garlands were taken on a procession in the four mada streets and later adorned to the presiding deity as a part of Goda Parinayam CELESTIAL MOCK HUNT:Here the annual celestial Utsavam was performed with religious pomp and gaiety with the processional deities of Lord Malayappa Swamy and Lord Srikrishna Swamy throwing the weapons at replica of wild animals in the green woods of Seschachala ranges and mock hunt was executed Temple priests and officials on behalf of Lord Malayappa and Lord Krishna Swamy participated in this celestial hunt festival on the auspicious Kanuma day The objective of the project is to contribute and complement Government efforts towards food self-sufficiency Project Manager for Kanuma Farato Kafo Rice Project saying part of the harvested rice would be given to the community while the rest would be sold to support women pay for their children’s school fees Dr Bassene expressed their plan to dig a borehole and fence the area noting that they are interested not only in making profit but also in supporting women of the area individuals and organisations to partner with them saying though prices for basic commodities are expensive in the country “such initiatives will help reduce” expenditure “Embracing agriculture is the solution,” he stated He also said that they aim to cultivate rice at the project site year-round as well as expand the project to other parts of the country thanked the alkalo and community of Kanuma for providing them with the land to cultivate rice to fight hunger and contribute to food self-sufficiency in the country a Gambian investor who is working with the Saudi and Turkey Governments The Star GSM CEO promised to donate a scanning machine to Essau District Hospital through Kanuma village developmental economics and philanthropist also commended women of Kanuma for their commitment and dedication to the project saying if they could sustain the project it would cut down the importation of rice in the country President of Kanuma Farato Kafo Rice Project thanked the investors and Star GSM for their support She added that government cannot do it all alone and therefore called on all and sundry to work with government in developing agriculture and ensure “we eat what we grow” Your browser does not support JavaScript, or it is disabled．Please check the site policy for more information National Report Tochigi Prefecture--A shipping tag addressed to a Japanese soldier killed in the Battle of Iwojima was returned to a bereaved family member 77 years after World War II measuring about 8 centimeters wide and 19 cm long who was killed in the battle fought in February and March 1945 “I heard that nothing came back from Iwoto,” said Noriko Kawarai “I think it’s a miracle to receive it in good condition like this.” The wooden tag contains the name of Murota’s father suggesting it was attached to a package he had sent to Murota when he was an apprentice officer of the 311th Independent Infantry Battalion of the Imperial Japanese Army The tag said the contents of the package were four books three packs of Seirogan anti-diarrheal pills “Everything was hard to come by at the time,” Noriko said “I think my grandfather did everything he could to get these items.” who was assigned to a garrison in the island’s western district was killed on March 17 when the Imperial Japanese Army launched an all-out attack against the U.S troops who had landed on the volcanic island The bloody battle claimed the lives of about 22,000 Japanese soldiers and around 7,000 U.S Navy member found the tag after landing on the island on March 25 the day before organized resistance by Japanese troops ended The American was dispatched there to work on construction of an airfield and other projects.He took the tag with him to the United States as a wartime memento his son found the tag while sorting through his father’s home The son donated the tag to a war museum in the U.S a writer and researcher of Pacific War history learned about the wooden tag and suggested returning the item to bereaved family members in Japan Kanuma Mayor Shin Sato received a letter from King Kanuma city officials found Mitsuko Kawarai saying it was “wonderful news” to learn that they found Murota’s bereaved family The tag was delivered to the Kanuma City Hall in October through King’s friend living in Kyoto Noriko had learned about her uncle from her grandmother and seen his photographs at her parents’ home She also read his diary and was impressed by his intellectual personality who used to describe how wonderful her brother was died in October before she had a chance to see the tag “I had thought it was only natural not to receive any personal items of my uncle because few people survived on Iwoto,” Noriko said "I had been told about the tragic stories from during the war I felt afresh that I should not forget them.” She said she would place the tag on the family Buddhist altar but intends to later entrust the item to a museum or other institution dedicated to passing down war memories Eruption of magma off Iwoto island seen as 1st in 1,000 years Pandemic delays maritime search for remains from Pacific War Volcanic Iwoto island rising at unmatched rate of 1 meter a year Dog tag of man killed in Battle of Okinawa given to daughter Ministry tells island city it will host SDF base Information on the latest cherry blossom conditions Please right click to use your browser’s translation function.) A series based on diplomatic documents declassified by Japan’s Foreign Ministry Here is a collection of first-hand accounts by “hibakusha” atomic bomb survivors chefs and others involved in the field of food introduce their special recipes intertwined with their paths in life A series about Japanese-Americans and their memories of World War II In-house News and Messages No reproduction or republication without written permission The decision to cut funding for SBC-103 came as part of a strategic evaluation and was tied to increases in development timelines and updated cash flows But it's not clear what changed in Alexion's development plans While the drug was still in early-stage testing SBC-103 had been seen as the leading pipeline candidate in the acquisition of Synageva which also brought the rare disease drug Kanuma (sebelipase alfa) on board Alexion executives painted the move as a prioritization decision pointing to continued enrollment in ALXN-1210 a potential complement to the company's principal revenue driver Soliris (eculizumab) SBC-103 had been granted both an Orphan Drug designation and Fast Track designation from the Food and Drug Administration. Designed to treat children with MPS IIIB (also known as Sanfilippo syndrome type b), the drug had shown some preliminary evidence of disease stabilization in trial results released last summer MPS IIIB is a progressive and serious genetic lysosomal storage disease Symptoms typically first emerge in infancy and more than half of children diagnosed with the condition die by age 17 Analysts from Jefferies noted uncertain neurocognitive effects could have played a role in the decision That drug is currently in Phase 3 testing for treatment-naive adult patients with paroxysmal nocturnal hemoglobinuria and could potentially reach the market by late 2018 or early 2019 But the decision to cut further funding for SBC-103 draws attention to Alexion's returns from its $8.4 billion deal to pick up Synageva earning only $11 million in sales in the fourth quarter last year as well as Alexion's other marketed drug Strensiq (asfotase alfa) has meant Alexion continues to be almost wholly dependent on revenues from Soliris Alexion has given guidance that predicts Soliris will account for nearly 90% of all revenues in 2017 Interim CEO David Brennan, who took over after an investigation into Soliris sales practices led to the departure of the former CEO and CFO can still hang his hat on a growing Soliris franchise But questions may begin to mount about the future especially as the search for a new permanent CEO continues Get the free daily newsletter read by industry experts Only five biotechs went public in the first quarter — and none have done so since early February — extending an IPO slump that's lasted over three years Striking study results last year indicated a new type of medicine may improve on Merck’s immunotherapy spurring a wave of research practically overnight The free newsletter covering the top industry headlines the temple administration of TTD has offered devotees to render “Go Seva” on the festival day of Kanuma in SV Goshala in Tirupati on Thursday TTD is contemplating to celebrate in a big way the “Go Mahotsavam” on the third day of Sankranthi festival on January 16 in Goshala in Tirupati the TTD has mulled an idea to involve devotees also to feed “Go Mata” with jaggery-rice mixed fodder with a noble aim to reinstate among them the importance of worship of cow as “Gomata” in Hindu Sanatana Dharma Meanwhile on this auspicious day of Kanuma the day starts with flute rendition by artistes by 6am followed by Veda Parayanam between 8am and 9am There will be bhajans by Dasa Sahitya Project artistes from 9am and 10:30am followed by special puja to Sri Venugopala Swamy temple located in the premises Later cultural programmes will follow till afternoon Japanese version