FDA approves Scholar Rock’s ISEMBYLD for spinal muscular atrophy patients aged 2 and older on SMN2 therapy

Phase 3 SAPPHIRE showed a 2.2-point HFMSE gain versus placebo at one year; 34.2% achieved a 3-point or greater improvement.

FDA cleared Scholar Rock’s ISEMBYLD (apitegromab-mstn) for spinal muscular atrophy in adults and children aged 2 and older on SMN2-targeted therapy.

The company said the product is expected to ship in the coming days as the U.S. launch gets underway.