Treatment with risdiplam was associated with improvement or maintenance of motor function in patients 2-25 years old with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA), according to 2-year data from Part 2 of the SUNFISH trial. The new research was presented at the 2021 Muscular Dystrophy Association Virtual Clinical & Scientific Conference.

SUNFISH (ClinicalTrials.gov: NCT02908685) is a randomized, double-blind, placebo-controlled study assessing the safety and efficacy of risdiplam, a survival of motor neuron 2 (SMN2) splicing modifier, in adult and pediatric patients with Type 2 and Type 3 SMA. In Part 2 of the trial, patients 2-25 years of age received either risdiplam for 24 months or placebo for 12 months followed by risdiplam for 12 months.

Findings at 24 months showed that treatment with risdiplam maintained motor function improvements (as measured by Motor Function Measure) and increased motor function (as measured by Revised Upper Limb Module and the Hammersmith Functional Motor Scale-Expanded) between months 12 and 24. Stabilized motor function was also observed in patients who started risdiplam treatment after 12 months of placebo. Moreover, improvements in self-reported and caregiver-reported independence were noted.

“These results build on the 1-year findings from the SUNFISH trial and importantly show the durability of improvement or stabilization of motor function through 2 years of treatment,” said SUNFISH principal investigator Eugenio Mercuri, MD, PhD, Department of Pediatric Neurology, Catholic University, Rome, Italy. “In addition, with no new safety signals identified, these second year results may support the favorable benefit-risk profile of [risdiplam] over a longer period of time.”


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In August 2020, the Food and Drug Administration approved risdiplam (Evrysdi™) for the treatment of SMA in adults and children 2 months of age and older. In addition to the SUNFISH study, the treatment is currently being evaluated in 3 other clinical trials: FIREFISH (ClinicalTrials.gov: NCT02913482), JEWELFISH (ClinicalTrials.gov: NCT03032172), and RAINBOWFISH (ClinicalTrials.gov: NCT03779334).

Reference

New 2-year data show Genentech’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 With Type 2 or Type 3 spinal muscular atrophy (SMA). [press release]. San Francisco, CA: Genentech; March 16, 2021.