The FDA has approved Scholar Rock’s ISEMBYLD for spinal muscular atrophy in adults and children ages 2 and older who are currently receiving a survival motor neuron 2-targeted treatment. ISEMBYLD is the first muscle-targeted treatment approved for children and adults with SMA who are receiving an SMN2-targeted treatment.
The approval was based on results from the Phase 3 SAPPHIRE study, according to a Sept. 11 press release. Patients receiving the recommended 10 milligram dose showed a 2.2-point improvement in motor function compared with placebo at one year.
The study also found 34.2% of ISEMBYLD-treated patients had a 3-point or greater increase in motor function compared with 13.5% of placebo-treated patients.
The safety database includes more than 500 individuals across apitegromab clinical studies globally, some of whom have been on treatment for more than seven years. Fractures occurred in 9% of patients treated with ISEMBYLD 10 milligram compared with 2% of those who received placebo.
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