Qalsody FDA Approval History
Last updated by Judith Stewart, BPharm on May 2, 2023.
FDA Approved: Yes (First approved April 25, 2023)
Brand name: Qalsody
Generic name: tofersen
Dosage form: Injection
Company: Biogen Inc.
Treatment for: Amyotrophic Lateral Sclerosis
Qalsody (tofersen) is an antisense oligonucleotide indicated 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 observed in patients treated with Qalsody. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).
- Amyotrophic lateral sclerosis (ALS) is a rare, progressive neurodegenerative disease that results in the loss of motor neurons in the brain and the spinal cord that are responsible for controlling voluntary muscle movement. Superoxide dismutase (SOD1) ALS is caused by mutations in superoxide dismutase 1 gene (SOD1). It is the second most common form of familial ALS, accounting for up to 20 percent of cases, and 2 percent of all ALS cases.
- Qalsody works by binding to SOD1 mRNA to reduce SOD1 protein production, potentially slowing the fatal progression of SOD1-ALS.
- Qalsody is administered by intrathecal injection every 14 days for three loading doses, then every 28 days thereafter.
- 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.
Development timeline for Qalsody
Further information
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