DIRS.INFO The most relevant business & tech news

☆ Insight: What happens when a $2 million gene therapy is not enough
▼ + stars: | 2023-08-12 | by ( Deena Beasley | ) www.reuters.com time to read: +13 min

Their experience raises broader questions around other high-cost gene therapies coming to market, sometimes after accelerated regulatory approvals, drug pricing experts said. Gene therapies work by replacing genes – the body's blueprint for its development. The gene Zolgensma delivers instructs the body to make a protein vital for muscle control. If gene therapies do fall short, it becomes harder to justify prices that researchers have argued are already poor value. More recently, the first hemophilia gene therapy approved by the U.S. Food and Drug Administration was priced by CSL Behring at $3.5 million; 26 more gene therapies are in late-stage development, according to IQVIA.

Persons: Elizabeth Kutschke, Ben, Zolgensma, Ben Kutschke, neurologists, Sitra Tauscher, Wisniewski, Ben's, Roger Hajjar, Brigham Gene, Kutschke, Vasant Narasimhan, Stacie Dusetzina, Roche's, Biogen, Roche, Maha Radhakrishnan, Steven Pearson, It's, Sree Chaguturu, Amanda Cook, Weston, Jackson, Cook, Elizabeth, Jerry Mendell, Russell Butterfield, –, Biogen's, Mendell, UMR, Spinraza, Eric Cox, Caroline Humer, Sara Ledwith Organizations: Reuters, U.S, Novartis, IQVIA Institute, Human Data, Novartis Gene Therapies, Mass, Cell Therapy, U.S . Food, Drug Administration, CSL Behring, CSL, Nashville's Vanderbilt University School of Medicine, Clinical, Economic, CVS Health, Aetna, SMA, Nationwide Children's Hospital, University of Utah Health, Children's, UnitedHealth, Thomson Locations: Oak Park, Berwyn , Illinois, Swiss, U.S, Lebanon , Virginia, United States, Columbus , Ohio, Russia, Kazakhstan, Chicago

☆ EMA seeks to issue guidance on liver damage from Novartis' gene therapy
▼ + stars: | 2023-01-13 | by ( ) www.reuters.com time to read: +1 min

Jan 13 (Reuters) - The European Medicines Agency (EMA) said on Friday it plans to issue advice for physicians using Novartis AG's (NOVN.S) Zolgensma, calling on them to monitor patients for any liver injury after treatment. The statement follows two deaths due to liver failure after treatment with the gene therapy against spinal muscular atrophy, reported by Novartis in August. Spinal muscular atrophy is a group of rare genetic disorders which affect the nerve cells and cause muscle wasting and weakness. The advice for healthcare professionals has yet to be approved by further expert panels at EMA, which typically takes a few weeks. In Europe, however, such letters had been the subject of discussions with EMA, but Novartis would soon be cleared to send them in EU member states, it added.

☆ FDA approves $3.5 million treatment for hemophilia, now the most expensive drug in the world
▼ + stars: | 2022-11-23 | by ( Deidre Mcphillips | ) edition.cnn.com time to read: +2 min

CNN —The US Food and Drug Administration on Tuesday approved Hemgenix, a new drug to treat hemophilia. Manufacturer CSL Behring set the price at $3.5 million per treatment, making it the most expensive drug in the world. In a recent cost-effectiveness analysis of the drug, weighing health benefits against offset costs, ICER suggested that a fair price for the drug to be between $2.93 million and $2.96 million. According to an analysis by GoodRx, the most expensive drug in the US previously was Zolgensma, which was approved by the FDA in 2019 to treat spinal muscular atrophy and priced at $2.1 million for a course of treatment. FDA approved Hemgenix based on safety and effectiveness evaluated in two studies of about 60 adult men.

Oct 25 (Reuters) - Biogen Inc (BIIB.O) on Tuesday said the Alzheimer's disease drug it is developing with Japan's Eisai Co Ltd (4523.T) would be used by doctors in a competitive market as investors wait for data from rival therapies made by Eli Lilly and Co (LLY.N) and Roche (ROG.S). The drug, lecanemab was shown to slow the progress of the disease by 27% in a trial last month, and additional data is expected next month. "The data needs to be seen from the other anti-amyloid therapies before we decide what is going to be meaningful," said Biogen interim research chief Priya Singhal. Its costs were about halved as the company significantly cut back on commercial infrastructure around the launch of Aduhelm, its previous Alzheimer's drug that has sputtered amid controversy over efficacy and lack of coverage by health insurers. read moreInvestors have been pinning hopes on the Alzheimer's drug as cheaper generics of Biogen's multiple sclerosis treatment Tecfidera have entered the market, hurting sales.

Register now for FREE unlimited access to Reuters.com RegisterSwiss drugmaker Novartis' logo is seen at the company's plant in the northern Swiss town of Stein, Switzerland October 23, 2017. Register now for FREE unlimited access to Reuters.com RegisterIt ranked 10th in the U.S. market last year, the company added. Novartis also said it would focus in future on five areas for investment: cardiovascular, immunology, neuroscience, solid tumors and hematology medicine. The company last month said it planned to spin off Sandoz to sharpen its focus on its patented prescription medicines. Register now for FREE unlimited access to Reuters.com RegisterEditing by Mark Potter and Jason NeelyOur Standards: The Thomson Reuters Trust Principles.

☆ Ученые разрабатывают назальный спрей против коронавируса COVID-19
▼ + stars: | 2020-12-03 | by ( Mld Media Srl | ) noi.md time to read: +2 min

Ученые из Пенсильванского университета и биотехнологическая фирма Regeneron изучают, можно ли использовать технологию, разработанную для генной терапии, для изготовления назального спрея, который предотвратит заражение новым коронавирусом, передает medikforum-ru.turbopages.org. «Преимущество нашего подхода в том, что для его эффективности не нужна компетентная иммунная система», - сказал AFP Джеймс Уилсон, профессор медицины в Пенсильвании, возглавляющий проект. В феврале правительство США связывалось с Уилсоном, чтобы узнать, могут ли он и его лаборатория использовать эту технологию против COVID-19. Исследователи надеются, что назальный спрей может быть впрыснут через ноздри, проникнет в эпителиальные клетки носа и захватит их белковые механизмы, чтобы они вырабатывали антитела к Регенерону. AAV вызывают лишь умеренный иммунный ответ, поэтому побочные эффекты могут быть менее серьезными, чем у передовых вакцин, которые работают, обучая иммунную систему распознавать ключевой белок вируса.

Persons: Джеймс Уилсон, Уилсон, Регенерон, Дональд Трамп Organizations: AFP, Пенсильванский университет Locations: Пенсильвания, США

☆ Oamenii de știință americani lucrează la un spray nazal împotriva COVID-19
▼ + stars: | 2020-12-02 | by ( Publika.Md - Aici Sunt Știrile | ) www.publika.md time to read: +5 min

Oamenii de știință de la Universitatea din Pennsylvania și firma de biotehnologie Regeneron investighează dacă tehnologia dezvoltată pentru terapia genică poate fi utilizată pentru a produce un spray nazal care va preveni infecția cu noul coronavirus. Wilson a fost contactat de guvernul SUA în februarie pentru a vedea dacă el și laboratorul său ar putea folosi tehnologia împotriva COVID-19. Deoarece coronavirusul intră în plămâni prin pasajul nazal, spray-ul ar putea opri infecția în urmele sale. Amintim că Universitatea din Birmingham (UK) a anunțat că a dezvoltat un spray nazal care protejează împotriva coronavirusului și îi împiedică transmisia. În același timp, dacă o persoană care a folosit spray-ul strănută sau tușește, scade riscul ca particule active de COVID-19 să ajungă în căile respiratorii ale altei persoane, explică oamenii de știință.

Persons: James Wilson, Penn, Wilson, american Donald Trump ., Regeneron, Liam Grover Organizations: Universitatea din, Universitatea Locations: Universitatea din Pennsylvania, SUA, american, Pennsylvania, Universitatea din Birmingham, UK

Search resuls for: "Zolgensma"

7 mentions found