Genentech/Roche Small Molecule

Risdiplam is an investigational SMN2 splicing modifier that is given daily by mouth or by g-tube as a liquid and distributes widely throughout the body. Risdiplam has been designed to help the SMN2 gene produce more SMN protein, as people with SMA have a reduced level of SMN protein. Risdiplam is being developed by Genentech/Roche, in collaboration with PTC Therapeutics and the SMA Foundation. Risdiplam was previously known as RG7916.

Ongoing Clinical Trials

FIREFISH

What is FIREFISH?

FIREFISH is a two-part study in babies aged 1 to 7 months with Type 1 SMA. It is an open label study, which means that all babies receive risdiplam and there is no placebo. The objective of Part 1 was to assess the safety and concentration of risdiplam in plasma at several different dose levels. 

Next steps for FIREFISH

Part 1 has been completed. Information from Part 1 has allowed us to confirm the dose of risdiplam to be investigated in Part 2, which will assess the safety and effiacy of risdiplam. Participants of Part 1 are receiving risdiplam and are still enrolled in FIREFISH as part of an open-label extension phase. 

Part 2 is underway and will assess the safety and efficacy of risdiplam, including an additional 40 babies between the ages of 1 and 7 months. The main analysis of efficacy and safety of risdiplam will occur after all babies have completed one year of treatment, and efficacy will be determined by the number of babies that can sit without assistance after one year of treatment.

SUNFISH

What is SUNFISH?

SUNFISH is a two-part study evaluating risdiplam in people with Type 2 and 3 SMA between 2 and 25 years of age. The objective of Part 1 was to assess the safety profile and concentration of risdiplam at several different dose levels as well as the level of SMN protein in blood in this population. Participants were randomized so that 2 out of every 3 people receive risdiplam and one receives placebo.

Next steps for SUNFISH

Information from Part 1 has allowed us to confirm the dose of risdiplam to be investigated in Part 2. Part 2 will assess the efficacy and safety of risdiplam. Enrollment into Part 2 of SUNFISH is almost complete, and approximately 168 participants will be included. The main analysis of the efficacy and safety of risdiplam will take place after all participants receive one year of treatment.

JEWELFISH

What is JEWELFISH?

JEWELFISH is an exploratory study investigating the safety of risdiplam in people who have Type 2 or 3 SMA, are between 12 and 60 years old and who have previously taken part in a clinical study with another SMN2 targeting therapy.

Next steps for JEWELFISH

The JEWELFISH study is currently ongoing in people aged 12 to 60 years who have previously received an SMN2 targeting therapy as part of a clinical study. We are in conversations with Health Authorities to broaden the inclusion criteria and increase the number of participants in JEWELFISH to include those who:

  • received nusinersen as part of their regular medical care for SMA
  • have SMA Type I
  • are 6 months of age or older. 

Further plans for JEWELFISH include eligibility for people who are still actively taking part in the OLEOS study (olesoxime long term safety and effiacacy study). These updates to JEWELFISH will be made subject to approval by Health Authorities and Ethics Committees. 

Upcoming Clinical Trials

RAINBOWFISH

The RAINBOWFISH study will evaluate risdiplam in pre-symptomatic babies. More details on this study will be available in the coming months.

Resources and Further Information

Data and Study Results 

© 2019 Cure SMA | Cure SMA is recognized as tax-exempt under section 501(c)(3) of the Internal Revenue Code. EIN: 36-3320440. | Privacy Policy

Powered by Blackbaud
nonprofit software