Tofersen: First Approval

Drugs. 2023 Jul;83(11):1039-1043. doi: 10.1007/s40265-023-01904-6.

Abstract

Tofersen (Qalsody™) is an antisense oligonucleotide being developed by Biogen for the treatment of amyotrophic lateral sclerosis (ALS). On 25 April 2023, tofersen was approved in the USA for the treatment of ALS in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This article summarizes the milestones in the development of tofersen leading to this first approval for ALS.

Publication types

  • Review

MeSH terms

  • Adult
  • Amyotrophic Lateral Sclerosis* / drug therapy
  • Amyotrophic Lateral Sclerosis* / genetics
  • Humans
  • Mutation
  • Oligonucleotides / pharmacology
  • Oligonucleotides / therapeutic use
  • Superoxide Dismutase / genetics
  • Superoxide Dismutase-1 / genetics

Substances

  • tofersen
  • Superoxide Dismutase
  • Oligonucleotides
  • Superoxide Dismutase-1