A comparative study of costs between nusinersen and onasemnogene abeparvovec

In association with the Novartis laboratory, which markets the onasemnogene abepavovec or OA (a gene therapy product prescribed for infantile spinal muscular atrophy linked to SMN1 or SMA), Dutch researchers have carried out a comparative study of the costs generated by innovative therapies (nusinersen versus OA):

  • the population studied was infants born in the Netherlands within the first year of life with type I SMA (with 2 or 3 copies of the SMN2 gene),
  • Several medico-economic models were used, comparing OA (single injection) and nusinersen (intrathecal injection every four months for life);
  • OA is less expensive than nusinersen from the 8th year of treatment,
  • Over a 20-year follow-up period, the difference would be between €1.5 and €2.9 million.

However interesting this research may be, it remains speculative, as little data is available on the long-term efficacy of these two drugs.

 

Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands. van der Schans S, Velikanova R, Weidlich D, et al. Eur J Health Econ. 2025 Feb.