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.
A lay explanation of what risdiplam is and the results obtained through Phase 1 clinical trials can be found here.
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
October 2021: Roche to start MANATEE, a global combination study in early 2022
July 2021: Data for Evrysdi® published in New England Journal of Medicine shows significant improvement in survival and motor milestones in babies with Type 1 SMA
June 2021: Evrysdi improves motor function in pre-symptomatic babies after one year and its safety profile in previously treated people with SMA is confirmed
March 2021: update on longterm data from part 2 of the SUNFISH trial
February 2021: EMA’s CHMP gives positive opinion for approval of Evrysdi™ (risdiplam) for the treatment of 5q SMA
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.
