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.
The first results of this second part, after a year of treatment, have just been communicated:
- 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 is expected by May 2020. A marketing authorization application should also be filed in 2020 in Europe.
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 »