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April 26, 2023April 25, 2023 - Biogen Inc. has announced 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.
Neurofilaments are proteins that are released from neurons when they are damaged, making them a marker of neurodegeneration.
Qalsody is the first approved treatment to target a genetic cause of ALS. Biogen collaborated with Ionis Pharmaceuticals on the early development of tofersen. Qalsody is administered by intrathecal injection every 14 days for three loading doses, then every 28 days thereafter.
Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s). The ongoing Phase 3 ATLAS study of tofersen in people with presymptomatic SOD1-ALS will serve as the confirmatory trial.
Warnings and precautions associated with Qalsody include myelitis and/ or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis. Common adverse reactions include pain, fatigue, arthralgia, cerebrospinal fluid white blood cell increased, and myalgia.
Biogen anticipates there may be variation in time to treatment as institutions and treatment centers learn about Qalsody.