Cohort Temporary Authorisation for Use for risdiplam in SMA type I, II and III

The ANSM (French National Agency for Medicines and Health Products Safety) has just granted risdiplam (Evrysdi) Cohort Temporary Authorisation for Use for “patients aged 2 months and over with a clinical diagnosis of SMA type I, II or III in case of treatment failure, intolerance or inability to administer treatment with respect to available alternative therapies, following an opinion from a French national FILNEMUS network multidisciplinary team (MDT) meeting. This authorisation will be effective from 6 April. Until this time, only individuals with SMA type I and II will be able to benefit from this treatment orally via Named Patient Temporary Authorisation for Use.


This decision is based on positive results obtained for risdiplam during the FIREFISH and SUNFISH trials. Based on these trials, a positive opinion has also been issued by the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP), which is recommending marketing authorisation for risdiplam in Europe, for individuals at least 2 months old with a clinical diagnosis of SMA type I, II or III or with 1 to 4 copies of SMN2. The European Commission must now decide on this European marketing authorisation (MA) within a period of around two months.


ANSM [online, in French]. Autorisation temporaire d’utilisation dite de cohorte – Protocole d’utilisation thérapeutique et de recueil d’informations Risdiplam 0,75 mg/mL, poudre pour solution buvable.  March 2021 version 1 [viewed 09/03/21]