Risdiplam: First Approval

Drugs. 2020 Nov;80(17):1853-1858. doi: 10.1007/s40265-020-01410-z.


Risdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein deficiency. The drug boosts the ability of an alternative gene SMN2 to produce full-length and functional SMN protein. In August 2020, Evrysdi™ (risdiplam) received its first approval in the USA for the treatment of spinal muscular atrophy in patients 2 months of age and older. Risdiplam is in pre-registration for this indication in numerous countries worldwide, including the European Union, Brazil, Chile, China, Indonesia, Russia, South Korea and Taiwan. This article summarizes the milestones in the development of risdiplam leading to this first approval for spinal muscular atrophy.

Publication types

  • Review

MeSH terms

  • Administration, Oral
  • Adolescent
  • Alternative Splicing / drug effects*
  • Azo Compounds / pharmacology
  • Azo Compounds / therapeutic use*
  • Child
  • Child, Preschool
  • Clinical Trials, Phase II as Topic
  • Clinical Trials, Phase III as Topic
  • Dose-Response Relationship, Drug
  • Drug Approval*
  • Humans
  • Infant
  • Multicenter Studies as Topic
  • Muscular Atrophy, Spinal / drug therapy*
  • Muscular Atrophy, Spinal / genetics
  • Mutation
  • Pyrimidines / pharmacology
  • Pyrimidines / therapeutic use*
  • Survival of Motor Neuron 2 Protein / deficiency
  • Survival of Motor Neuron 2 Protein / genetics
  • United States
  • United States Food and Drug Administration / legislation & jurisprudence
  • Young Adult


  • Azo Compounds
  • Pyrimidines
  • SMN2 protein, human
  • Survival of Motor Neuron 2 Protein
  • Risdiplam