Risdiplam (or Evrysdi®) is a small drug molecule administered orally that corrects SMN2 gene splicing, thus producing the missing SMN1 protein in SMN1-related proximal spinal muscular atrophy (SMA). It has had marketing authorisation in the United States since August 2020. Following this, it received marketing authorisation in Brazil, Chile, South Korea, Georgia, Russia and Ukraine. To date, more than 2500 patients have received risdiplam worldwide.
CHMP gives green light
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has just issued its opinion regarding risdiplam; it recommends granting marketing authorisation for patients aged over two months with a clinical diagnosis of SMA type I, II or III, or with one to four copies of SMN2. The European Commission will confirm, or not confirm, this opinion within a period of approximately two months.
The first part of the FIREFISH trial shows positive results at 12 months
This opinion is based on motor function improvement obtained during the SUNFISH trial (among patients with SMA type II or III, aged 2 to 25 years) and the FIREFISH trial (among symptomatic newborns with SMA type I, aged 2 to 7 months). These two trials, which were also conducted in France, each consist of two parts, the first part to evaluate safety and find the optimum product dose, and the second part to test the efficacy of the optimum dose selected in the first part.
The results at 12 months for the first part of the FIREFISH trial, relating to 21 newborns, have just been published:
- 90% of the newborns are not on permanent ventilation,
- none of them has swallowing problems,
- one third of them are able to stay seated independently for at least 5 seconds,
- Risdiplam increases functional SMN protein blood levels.
« First oral treatment for spinal muscular atrophy (SMA) recommended for approval », EMA, 26 février 2021 [Site internet de l’EMA consulté le 01/03/21]
« Roche announces results from Evrysdi (risdiplam) study in infants with Type 1 spinal muscular atrophy (SMA) published in New England Journal of Medicine » Roche, Communiqué de presse du 25 février 2021