SMA, that had no long-term treatment so far, has experienced an unprecedented therapeutic revolution in recent years: an antisense oligonucleotide (nusinersen or Spinraza®), a gene therapy product (onasemnogene abeparvovec or Zolgensma ®) and now an oral treatment, risdiplam (Evrysdi ™), now constitutes an innovative therapeutic arsenal for patients with SMA.
In fact, the European Commission has just granted a European Marketing Authorization for risdiplam for “patients aged over two months, with a clinical diagnosis of type I, type II or type III SMA or with one to four copies of the SMN2 gene ”. It concerns the 27 Member States of the European Union, as well as Iceland, Liechtenstein and Norway.
The risdiplam, currently prescribed in France via a nominative Temporary Use Authorization for use (ATU), could be prescribed at the beginning of April via a cohort ATU and then as part of a post-ATU device pending its commercialization.
This decision is based in particular on the improvement in motor function obtained during the SUNFISH and FIREFISH trials, which gave rise to a positive opinion from the Committee for Medicines for Human Use (CHMP) of the European Medicines Agency last February. To date, more than 3000 patients have been treated with risdiplam worldwide.
Roche’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy. Communiqué de presse du 30 mars 2021. Roche