How flexible should the U.S. FDA evidentiary standards be for a therapy addressing a significant unmet need in a disease such as amyotrophic lateral sclerosis (ALS)? That’s the question the agency’s Cellular, Tissue and Gene Therapies Advisory Committee will ponder Sept. 27 as it looks at the data for Brainstorm Cell Therapuetics Inc.’s Nurown (debamestrocel), a mesenchymal stromal cell therapy targeting ALS.
The Biogen amyotrophic lateral sclerosis drug, Qalsody, treats patients whose disease is driven by mutations to a gene called SOD1. It’s the first drug approved to target a genetic cause of ALS.
Biogen Inc. (BIIB) said that the U.S. Food and Drug Administration has approved Qalsody (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis or ALS in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.