Roche’s small molecule, risdiplam (RG7916), is an orally-available compound developed in collaboration with the SMA Foundation and PTC Therapeutics and which is used to increase the amount of SMN protein made by the SMN2 gene.
There are currently 4 trials for risdiplam, which started in December 2016 in SMA Types 1, 2 & 3:
| ||Study Name||SMA Population||Age
|FIREFISH||Type 1||Between 1 & 7 months
|SUNFISH||Type 2 & 3||Between 2 & 25 years
|JEWELFISH||Type 1, 2 & 3||Between 6 months & 60 years and has received nusinersen or participated in trial of other SMN-targeting therapy or olesoxime
|RAINBOWFISH||Presymptomatic||Up to 6 weeks
A lay explanation of the results obtained through Phase 1 clinical trials can be found below:
Roche has a dedicated website for these SMA trials, where more information can be found.
- January 2020: Roche announces global pre-approval access/ compassionate use (PAA/CU) plans for risdiplam
- November 2019: FDA grants priority review to risdiplam for the treatment of SMA & Roche’s letter to the community
- November 2019: Roche’s risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 SMA
- May 2019: Update on FIREFISH & SUNFISH presented at the 2019 AAN conference
- October 2018: Update of FIREFISH & SUNFISH studies of risdiplam
- March 2018: Start of pivotal part of FIREFISH for babies with Type 1 SMA
- October 2017: Interim results of Part 1 of the SUNFISH study indicate RG7916 is well tolerated at all doses. The first patient has now been enrolled for Part 2.
- September 2017: Start of Part 2 of the SUNFISH study. Enrolment is expected to begin in September 2017 in France and Belgium. Italy, Switzerland, Germany and Spain will begin later in the year.
- August 2017: Eye toxicity finding in RG7800 and its relationship with RG7916.
Clinical trials registers: