FDA Accelerates Approval for Qalsody, the First Treatment Targeting Genetic Cause of ALS

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Biogen Inc. announced the approval, a groundbreaking treatment for adults with ALS, offering hope to patients suffering from SOD1-ALS, a rare and fatal genetic form of the disease.

Qalsody is considered a major scientific advancement, the first approved treatment to target a genetic cause of ALS. Developed in collaboration with Ionis Pharmaceuticals, it has demonstrated a significant reduction in the decline of clinical function, respiratory strength, and muscle strength in patients with SOD1-ALS. The treatment will be available for shipment to healthcare providers in the U.S. within a week, offering hope for an improved quality of life for those living with this rare and devastating form of ALS.

“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.”


Reference: 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 | April 25, 2023 | Biogen