Tofersen (1 brand name, 1 dosage form, 1 route)
Available dosage forms: SOLUTION.
Routes of administration: INTRATHECAL.
Brand Names
- QALSODY
Indications and Usage
QALSODY is 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 (NfL) observed in patients treated with QALSODY [see Clinical Studies (14)]. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).
Contraindications
None.
Adverse Reactions
The following clinically significant adverse reactions are discussed elsewhere in the labeling: Myelitis and/or Radiculitis [see Warnings and Precautions (5.1)] Papilledema and Elevated Intracranial Pressure [see Warnings and Precautions (5.2)] Aseptic Meningitis [see Warnings and Precautions (5.3)]
Mechanism of Action
Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.