The primary endpoint of the second part of the FIREFISH clinical trial has been met. This is an announcement by Roche and PTC Therapeutics, the two laboratories that develop risdiplam, a small molecule orally administered correcting the maturation of SMN2.
The FIREFISH clinical trial is an open-label, international trial (also taking place in France), involving 62 infants with SMA type 1, 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 efficacy of the product over two years.
An improvement over 12 months…
En avril 2020, les premiers résultats de cette seconde partie, après un an de traitement, étaient communiqués :
In April 2020, the first results of this second part, after a year of treatment, were reported:
- 29 % infants are able to sit unassisted for 5 seconds (primary endpoint),
- 44% can hold their head upright,
- 32% manage to roll sideways,
- 5% stand with support,
- 95% are able to swallow,
- 89% can eat orally,
- 93% are still alive,
- 85% have not had intercurrent medical events.
These encouraging results, in addition to the results of the second part of the SUNFISH trial, conducted in SMA type 2 and 3, will support the request for marketing authorization for risdisplam from the FDA whose response was expected by May 2020. A marketing authorization application was also to be filed in 2020 in Europe.
… which is confirmed at 24 months
After one year of treatment with risdiplam (Evrysdi), the primary endpoint of Part 2 of the FIREFISH trial was met. And the improvements achieved last up to two years of treatment. This is what Roche and PTC Therapeutics announced, the two laboratories developing risdiplam, a small molecule administered orally that corrects the maturation of SMN2.
The FIREFISH trial is an open-label, international trial (also taking place in France), involving 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. The first results of this second part, after two years of treatment, have just been communicated:
- 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 a support.
Access Roche press release (28 April 2020): « Roche’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA) »
Access PTC Therapeutics press release (28 April 2020): « Confirmatory Part 2 of FIREFISH Demonstrates Survival and Motor Milestones Not Seen in Natural History in Infants with Type 1 Spinal Muscular Atrophy »
Access Roche press release (15 April 2021): « Roche’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA) »
Access PTC Therapeutics press release (15 April 2021): « Continued Improvements in Motor Milestones in Infants with Type 1 Spinal Muscular Atrophy After Two Years of Treatment with Evrysdi™ »
