Biopharmaceutical company Scholar Rock’s Isembyld treatment was approved by the FDA to treat spinal muscular atrophy in adults and pediatric patients 2 years of age and older who are currently receiving a survival motor neuron 2-targeted treatment.
Isembyld is the first and only muscle-targeted treatment for this population, according to the Sept. 11 news release. The treatment directly targets the muscular component of SMA.
The approval was based on results from the Phase 3 Sapphire study. Patients receiving Isembyld 10 mg/kg and an SMN2-targeted treatment demonstrated a 2.2-point improvement in the Hammersmith Functional Motor Scale-Expanded compared with patients receiving an SMN2-targeted treatment alone at one year.
Additionally, 34.2% of Isembyld-treated patients showed a ≥ 3-point increase in HFMSE compared with 13.5% of placebo-treated patients.
Isembyld will be available to ship in the coming days.
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