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 RAINBOWFISH Presymptomatic Up to 6 weeks
Roche has a
dedicated website for these SMA trials, where more information can be found.
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 December 2020: Summary of work carried out in 2020 together with lay summaries on FIREFISH & SUNFISH September 2020: Update on part 1 of the FIREFISH clinical trial August 2020: EMA has validated the Marketing Authorisation Application (MAA) for risdiplam for the proposed use in people living with SMA August 2020: Risdiplam approved for the treatment of SMA in the US June 2020: Roche presents new data on SUNFISH & JEWELFISH trials at the Cure SMA Annual 2020 virtual Conference April 2020: Update on the clinical development of risdiplam February 2020: Roche completes recruitment for JEWELFISH clinical trial February 2020: Risdiplam shown to improve motor function in people aged 2 to 25 with type 2 or 3 SMA January 2020: Risdiplam’s FIREFISH meets primary endpoint in trial in infants with type 1 SMA 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: