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FDA Grants Accelerated Approval for QALSODY™ (tofersen) for SOD1-ALS, a Major Scientific Advancement as the First Treatment to Target a Genetic Cause of ALS
FDA granted accelerated approval of QALSODY based on a reduction of neurofilament, a marker of neurodegeneration1Superoxide dismutase 1 (SOD1)-amyotrophic
About this update from Biogen Inc.
[{"type":"text","content":"FDA granted accelerated approval of QALSODY based on a reduction of neurofilament, a marker of neurodegeneration1Superoxide dismutase 1 (SOD1)-amyotrophic lateral sclerosis (ALS) is a devastating, uniformly fatal,2 and ultra-rare genetic form of ALS3-4 with approximately 330 people in the U.S. living with the disease5 CAMBRIDGE, Mass., April 25, 2023 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) announced today that the U.S. Food and Drug Administration (FDA) has approved QALSODY™ (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).1 The ongoing Phase 3 ATLAS study of tofersen in people with presymptomatic SOD1-ALS will serve as the confirmatory trial.1 Neurofilaments are proteins that are released from neurons when they are damaged, making them a marker of neurodegeneration.6 “For more than a decade, Biogen has been steadfast in our commitment to pursuing treatments for ALS, and I want to thank the scientists as well as the entire ALS community who have all worked tirelessly to bring this first-of-its-kind treatment to people with SOD1-ALS,” said Christopher A. Viehbacher, President and Chief Executive Officer of Biogen. “Today also marks a pivotal moment in ALS research as we gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS. We believe this important scientific advancement will further accelerate innovative drug development for ALS.” QALSODY is the first approved treatment to target a genetic cause of ALS.1 Biogen collaborated with Ionis Pharmaceuticals on the early development of tofersen. Warnings and precautions associated with QALSODY were serious neurologic events, including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis. If symptoms consistent with myelitis, radiculitis papilledema, elevated intracranial, or aseptic meningitis develop, diagnostic workup and treatment should be initiated ...