regulatory
confidence high
sentiment positive
materiality 0.90
FDA approves Scholar Rock's ISEMBYLD for SMA; company clarifies post-marketing safety commitments
Scholar Rock Holding Corp
- FDA approved ISEMBYLD (apitegromab-mstn) for spinal muscular atrophy in adults/children 2+ on SMN2-targeted treatment.
- Corrects prior statement: FDA requires a pregnancy safety study and 15-day fracture alert reports as post-marketing commitments.
- Scholar Rock will conduct the study and submit fracture reports in compliance with FDA's request.