After one year of treatment with risdiplam (Evrysdi), the primary endpoint of the 2nd part of the FIREFISH trial was met. The improvements achieved last up to two years, according to Roche and PTC Therapeutics, two laboratories that are developing risdiplam, a small molecule administered orally which corrects the maturation of SMN2.
The FIREFISH study is an open-label, international trial (also taking place in France) and involves 62 infants with SMA aged 1 to 7 months. It takes place in two parts: the first lasting one month has shown that risdiplam is well tolerated; the second, still in progress, evaluates the effectiveness of the product over two years. Results of this second part, after two years of treatment, were reported:
- the lifespan of the participants increases,
- the need for permanent ventilation decreases,
- 61% of infants are able to sit up unassisted for 5 seconds,
- 63% of infants can keep their head upright,
- 15% of infants stand upright with support.
« Roche’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA) » Laboratoire Roche. Communiqué de presse du 15 avril 2021
« Continued Improvements in Motor Milestones in Infants with Type 1 Spinal Muscular Atrophy After Two Years of Treatment with Evrysdi™ »PTC Therapeutics. Communiqué de presse du 15 avril 2021
