An application for marketing authorisation for risdisplam (RO7034067 ou RG7916), in the treatment of SMN1-related proximal spinal muscular atrophy (SMA) was submitted to the FDA (US Food and Drug Administration), by Roche and PTC Therapeutics, who are developing the product. Risdiplam is a small drug molecule administered orally that corrects SMN2 maturation to produce the SMN protein that is missing in SMA.
This application is based on:
positive results obtained over 12 months in the first parts of the SUNFISH and FIREFISH trials: improvement in motor function in type 1, 2 and 3 SMA, without side effects;- preliminary results from part 2 of the SUNFISH trial, confirming an improvement in motor strength in type 2 and 3 SMA.
Given that this application has been granted priority review status (reducing the time-frame from 10 to 6 months), the FDA should give an opinion before 24 May 2020. If this application is approved, it will be the first oral treatment for SMA.
Access Roche press release (25/11/19) « FDA grants priority review to Roche’s risdiplam for spinal muscular atrophy »
Access PTC Therapeutics press release (25/11/19) « PTC Therapeutics Announces FDA Grants Priority Review to Risdiplam for the Treatment of Spinal Muscular Atrophy »
