SMA Press
AveXis Announces Single-Arm Design for U.S. Pivotal Study of AVXS-101 in SMA Type 1 Patients
CHICAGO, Nov. 01, 2016 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the planned pivotal study of AVXS-101 in spinal muscular atrophy (SMA) Type 1 will reflect a single-arm design, using natural history of the disease as…
Read MoreBiogen’s Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA
Biogen today announced that its New Drug Application (NDA) for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), has been accepted by the U.S. Food and Drug Administration (FDA) for Priority Review, and that the company’s Marketing Authorization Application (MAA) has been validated by the European Medicines Agency (EMA). Nusinersen had previously been granted…
Read MoreBiogen Submits NDA to the FDA for Nusinersen
Biogen and Ionis have taken a crucial step in the pathway to approval of nusinersen. Biogen has completed the submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) and will submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the coming weeks. Biogen has also applied…
Read MoreRoche Initiates New Phase 2 Clinical Trials
Roche has announced that the company is initiating two new Phase 2 studies in patients with Spinal Muscular Atrophy (SMA) to evaluate RG7916—SUNFISH and FIREFISH. RG7916 is an orally available drug that directly targets the underlying molecular deficiency of SMA by modulating SMN2 splicing to increase expression of full-length SMN2 mRNA and SMN protein from…
Read MoreBloomberg Article: New Hope for Kids With a Rare Disease, Thanks to a Dinner Party
Since grade school, Lauren Gibbs had been a dogged defensive player on her wheelchair-basketball team. Offense wasn’t a big part of her game because it was a struggle to heave the ball high enough to hit the rim. Then Gibbs, who was born with spinal muscular atrophy, enrolled in a trial of an experimental drug…
Read MoreBiogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy
CAMBRIDGE, Mass. & CARLSBAD, Calif.–Aug. 1, 2016– Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) today announced that nusinersen, their investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint pre-specified for the interim analysis of ENDEAR, the Phase 3 trial evaluating nusinersen in infantile-onset (consistent with Type 1) SMA. The analysis found that infants receiving…
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