[Risdiplam in spinal muscular atrophy]

Mengarelli C, Pichon-Riviere A, Alfie V, Argento F, García Martí S, Ciapponi A, Bardach A, Augustovski F, Alcaraz A
Record ID 32018002321
Original Title: Risdiplam en atrofia muscular espinal
Authors' recommendations: Moderate-quality evidence from preliminary data obtained from ongoing unpublished studies conducted in 2 to 7 months old children with type 1 spinal muscular atrophy, shows that oral risdiplam might improve survival, muscular skills (sitting down) and oral feeding after a two-year follow up when compared with placebo. Moderate-quality evidence from preliminary data obtained from a clinical trial, not published yet, conducted in patients with type II and type III spinal muscular atrophy shows that the use of risdiplam might improve motor skills (sitting down, swallowing, eating) when compared with placebo. No studies comparing risdiplam with other disease-modifying treatments such as nusinersen and onasemnogene abeparvovec have been found. No clinical practice guidelines recommending the use of risdiplam have been found despite having been approved by regulatory agencies from the US and Europe. US health funders authorize it in children 2 to 7 months old with specific genetic diagnosis of two or more copies of genes (type 1 spinal muscular atrophy) and in patients 2 months to 25 years old with three or more copies of genes (type II-III spinal muscular atrophy).The United Kingdom does not cover this drug because it is not considered cost-effective. Based on current international prices, it is unlikely that risdiplam becomes a cost-effective intervention in Argentina and it would have a high budget impact.
Project Status: Completed
Year Published: 2021
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Argentina
MeSH Terms
  • Spinal Muscular Atrophies of Childhood
  • Azo Compounds
  • Pyrimidines
  • Infant
  • Neuromuscular Agents
Organisation Name: Institute for Clinical Effectiveness and Health Policy
Contact Address: Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA
Contact Name: info@iecs.org.ar
Contact Email: info@iecs.org.ar
Copyright: Institute for Clinical Effectiveness and Health Policy (IECS)
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