Biogen Submits NDA for Nusinersen to Treat Spinal Muscular Atrophy
Sept. 26, 2016
Background: Biogen and Ionis Pharmaceuticals today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA).
Submission of the NDA is the first step toward getting approval of nusinersen to treat SMA in the United States. The FDA now has 60 days to either accept or reject the NDA. If accepted, the Agency will begin review of the application, a process that typically takes 10 months. However, Biogen has applied to receive Priority Review which, if granted, would shorten the review period for the drug from 10 months to six.
Today’s development follows news in August that showed encouraging interim results from the late-stage ENDEAR clinical trial to test nusinersen in infants with type 1 SMA. Based on those results, investigators stopped the trial to allow all participants to transition into an open-label trial called SHINE, in which all infants are being treated with the drug.
MDA has funded foundational work in SMA and invested nearly $750,000 in awards to Adrian Krainer at Cold Spring Harbor Laboratory in New York for early-stage development of nusinersen. Since its inception, MDA has invested more than $45 million in SMA research.
Statement from MDA Scientific Program Officer Amanda Haidet-Phillips, Ph.D.: “Today we mark an exciting step forward for our families living with spinal muscular atrophy as nusinersen formally moves toward FDA review. It’s an encouraging sign of progress and hope for all of us in the SMA community who have worked together to develop an effective treatment for this life-threatening disease.
“MDA is proud to have been part of early-stage research that helped lay the groundwork for nusinersen’s development, and our sense of urgency has never been greater as we move closer to our goal of helping all our families with SMA and related neuromuscular diseases.”
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at mda.org.
Roxan Triolo Olivas
MDA Vice President
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Spinal Muscular Atrophy News
- From Apples to Advocacy- A Quest ArticleJuly 1, 2017
- MDA Celebrates FDA Approval of Spinraza for Treatment of Spinal Muscular AtrophyDecember 23, 2016
- Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study in Later-Onset Spinal Muscular AtrophyNovember 7, 2016
- Biogen’s Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMAOctober 28, 2016
- Biogen Submits NDA for Nusinersen to Treat Spinal Muscular AtrophySeptember 27, 2016