SMA Newsroom

Nov 12, 2019

Roche's risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 SMA

Roche has announced positive data from the pivotal Part 2 of SUNFISH, a study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 SMA:

  • The study met its primary endpoint of change from baseline in a motor function test (the Motor Function Measure 32 - MFM-32 scale) after one year of treatment with risdiplam, compared to placebo.
  • No treatment related safety findings leading to study withdrawal have been seen in any risdiplam trial to date. Safety for risdiplam was consistent with its known safety profile and no new safety signals were identified.

“The positive outcome of this trial is an important milestone for people with Type 2 or 3 SMA, too many of whom remain untreated,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “SUNFISH is the largest placebo-controlled study ever undertaken in Type 2 or 3 SMA patients. We thank the SMA community for their partnership and look forward to sharing these results with regulators and bringing risdiplam to people living with this condition.”

SMA Europe is happy that risdiplam meets the primary endpoint for people with type 2 and 3 SMA so that treatment options will be available to our community soon. SMA Europe has a dedicated Patient Advisory Group (PAG) that has worked closely with the company to ensure that patients all over Europe will gain access as soon as possible. We are hoping to be able to share a Q&A very soon.

For more information

  • About risdiplam:
    • Risdiplam is an investigational survival motor neuron-2 (SMN2) splicing modifier, designed to durably increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. Roche leads the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics. Data from the SUNFISH study will be presented at an upcoming medical congress.
    • Risdiplam is being studied in a broad clinical trial programme in SMA, with patients ranging from birth to 60 years old, and includes patients previously treated with SMA-targeting therapies. The clinical trial population represents the broad real-world spectrum of people living with this disease with the aim of ensuring access for all appropriate patients.
  • About the SUNFISH Study: SUNFISH is a two part, double-blind, placebo controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA.