Risdiplam: a third medicine authorised in the United States for type 1, 2 and 3 SMA, for children 2 months of age or older and for adults of all ages

On 7 August 2020, Roche/Genentech PTC Therapeutics announced that risdiplam had been granted marketing authorisation for type 1, 2 and 3 SMA in the United States, under the commercial name of Evrysdi™. Evrysdi™ is an oral treatment that can be administered from the age of 2 months and to all ages, regardless of disease progression. A marketing authorisation application should be filed very soon with the EMA.

In France, Evrysdi™ can be prescribed for type 1 SMA in the context of a Temporary Authorisation for Use for a named patient. It is expected that Temporary Authorisation for Use will be opened up to other SMA types.

Efficacy with respect to motor function and respiratory function
The first results of the SUNFISH trial in type 2 or 3 SMA among patients 2 to 25 years of age (trial versus placebo), and the FIREFISH trial in type I SMA among infants 2 to 7 months of age, show:

  • among infants (FIREFISH trial): an improvement in motor function, in particular the ability of infants to sit without help at 12 months of treatment and effective respiratory function, allowing the life expectancy of the majority of the infants included in the trial to be increased beyond 15 months of age, without the need for ventilation;
  • among older children and adults (SUNFISH trial), an improvement in motor function at 12 months of treatment compared to untreated patients (taking the placebo).
  • Risdiplam was well tolerated in both trials.

Other ongoing trials
Risdiplam continues to be evaluated among representative SMA patients (in terms of age, SMA type, disease progression), with four ongoing trials:

  • the FIREFISH and SUNFISH trials, which are continuing;
  • the JEWELFISH trial in patients 6 months to 60 years of age who have already been treated with nusinersen (Spinraza®), olesoxime or AVXS-101 (Zolgensma®);
  • the RAINBOWFISH trial in infants less than 6 weeks of age who have not yet developed symptoms, but who have been diagnosed with SMA.


Access press releases, Aug. 7, 2020 :
Roche/ Genentech : « FDA Approves Genentech’s Evrysdi™ (risdiplam) for Treatment of Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older »

PTC Therapeutics : « PTC Therapeutics Announces FDA Approval of Evrysdi™ (risdiplam) for the Treatment of Spinal Muscular Atrophy in Adults and Children 2 months and older »