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Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings

Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings

Today, Biogen and Ionis Pharmaceuticals provided a statement regarding the decision to submit the first ever SMA regulatory filings for FDA approval. Dear Members of the SMA Community, Today marks an important day in our collective pursuit of a meaningful treatment for Spinal Muscular Atrophy (SMA). We are pleased to share the exciting news that we have reported positive results from a pre-specified interim analysis of the ENDEAR study in infants with Spinal Muscular Atrophy (SMA). As a result, plans are underway to transition all ENDEAR study participants to nusinersen in an open-label extension study and close the ENDEAR study. We plan to submit regulatory filings globally in the coming months. This step will hopefully bring us closer to our ultimate goal of making nusinersen available as quickly as possible as an approved therapy.

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