The SUNFISH (NCT02908685) pivotal part 2 study showed improved motor function after treatment with risdiplam for people with spinal muscular atrophy (SMA). The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after 1 year of treatment with risdiplam, compared to placebo.
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.
“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, MD, PhD, 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.”
Risdiplam is being studied in a broad clinical trial program in SMA, with participants ranging from newborns to 60 years old, and includes participants 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 participants.
Melissa W. Ko, MD; Kevin E. Lai, MD; and Devin D. Mackay, MD
Vanessa Baute Penry, MD; Rachana Gandhi Mehta, MD; and Fatemeh Sadeghifar, BS
Stephen M. Gollomp, MD, and Paul G. Mathew, MD, DNBPAS, FAAN, FAHS