[Onasemnogene abeparvovec (Zolgensma®) for spinal muscular atrophy]

Argento F, García Martí S, Colaci C, Alfie V, Ciapponi A, Bardach A, Augustovski F, Alcaraz A, Pichon-Riviere A
Record ID 32018004409
Original Title: Onasemnogene abeparvovec (Zolgensma®) en atrofia muscular espinal
Authors' recommendations: Low‐quality evidence suggests that onasemnogene abeparvovec may result in an increased net benefit in providing permanent mechanical ventilation‐free survival and improvement in motor milestones in children six months old or younger with Type I Spinal Muscular Atrophy (SPA), with no severe condition (not requiring permanent feeding support or mechanical ventilation), and with a biallelic mutation when compared with supportive care. Low‐quality indirect evidence, suggests that onasemnogene abeparvovec may improve permanent mechanical ventilation‐free survival in motor milestones when compared with nursinersen in this population. Very low‐quality evidence does not allow to draw conclusions about the treatment effectiveness in children over six months old. Clinical practice guidelines from Spain, Europe and Germany are not conclusive in recommending the use of onasemnogene abeparvovec or not; however, they make recommendations for its administration and follow up. One clinical practice guideline from the “Prof. Dr. Juan P. Garraham” Hospital in Argentina, considers this technology as an emerging one for SMA, but no recommendations are made. It is worth mentioning that this guideline was published very close to the date onasemnogene abeparvovec was approved by the United States Food and Drug Administration. Coverage policies from Latin America, Australia and the United States (public system) do not mention it. Canada and the United Kingdom cover it based on a price agreement. Canada covers it in six‐month old minors only and the United Kingdom in seven‐month old minors indicating the need for assessment by a national multidisciplinary team in children seven to 12 months old. Coverage policies from private United States funders cover onasemnogene abeparvovec in Type I SMA in children less than two years old meeting certain requirements. No cost‐effectiveness studies have been found in Argentina. Based on the budget impact estimated in the country, it is considered a very expensive treatment. Data from economic evaluations from other regions in the world suggest that it would not be cost‐effective in Argentina.
Project Status: Completed
Year Published: 2022
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Argentina
MeSH Terms
  • Spinal Muscular Atrophies of Childhood
  • Muscular Atrophy, Spinal
  • Genetic Therapy
  • Biological Products
  • Recombinant Fusion Proteins
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)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.