Risdiplam authorized in the US can now be prescribed in France in SMA type 2 under nominative TUA

On August 7, 2020, Roche/Genentech and PTC Therapeutics announced the market authorization for risdiplam (Evrysdi™) in SMA type 1, 2 and 3, in the United States. This oral treatment can be administered from the age of 2 months and at all ages, regardless of the course of the disease.

  • In France, nominative TUAs allow to prescribe Evrysdi™ to patients with type 2 SMA, as it was already the case for those with SMA type 1.
  • In addition, the European Medicines Agency (EMA) approved risdiplam . Its evaluation by the CHMP can begin and should last approximately 9 months.