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The Curtain Has Risen on the Competition for the First Generic Drug, so What Other Innovative Drugs in the Rare Disease Field Can We Look Forward to?
Xiaobin
January 23, 2025
The CDE website shows Qilu Pharmaceutical applied for the marketing of nusinersen generic. Once controversial for high price, it treats SMA. Now many firms are involved, and SMA treatment progresses.
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Reverse Mergers Among Chinese Pharma Firms are Rising
Xiaobin
January 23, 2025
Qilu Pharmaceutical applied for marketing of nusinersen injection, becoming the second in China. Nusinersen has a complex history due to high price. SMA treatment has multiple drugs and Chinese firms are making progress, with unmet needs remaining.
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Biogen Exercises Option for Exclusive License to BIIB115
contractpharma
January 04, 2022
BIIB115 is an investigational antisense oligonucleotide (ASO) in development for spinal muscular atrophy.
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China increases accessibility to rare disease treatment
NMPA/Xinhua
December 24, 2021
Two-and-half-year-old Xuanxuan survived a rare kidney disease in early December, thanks to fast-track approval of the transplant.
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Roche awarded 2022 Drug Discovery of the Year for Evrysdi®
EuropeanPharmaceuticalReview
December 07, 2021
The British Pharmacological Society has given the team of scientists who discovered and developed Evrysdi® (risdiplam) the Drug Discovery of the Year Award 2022.
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Novartis Announces New Phase 3 Study for Spinal Muscular Atrophy Treatment in Elderly Patients
americanpharmaceuticalreview
August 04, 2021
Novartis announced that the U.S. FDA determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019.
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Roche’s Evrysdi significantly improves lives of babies with SMA
europeanpharmaceuticalreview
August 02, 2021
The Phase II study of Evrysdi showed significant improvements in survival and motor milestones in babies with type 1 spinal muscular atrophy (SMA).
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Expanded access for Zolgensma following final NICE guidance
pharmatimes
July 12, 2021
The National Institute for Health and Care Excellence (NICE) has published its final guidance on Novartis’ spinal muscular atrophy (SMA) gene therapy Zolgensma.
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Zolgensma shows promise in presymptomatic SMA patients
pharmatimes
June 22, 2021
Novartis’ gene therapy Zolgensma has shown benefit for presymptomatic spinal muscular atrophy (SMA) patients, according to new data presented at the annual European Academy for Neurology (EAN) virtual congress.
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NICE draft guidance does not recommend Evrysdi for SMA
pharmatimes
June 04, 2021
The UK National Institute for Health and Care Excellence (NICE) has announced that it does not recommend Roche’s Evrysdi for the treatment of spinal muscular atrophy (SMA) in draft guidance.