Regulatory Event: USFDA Biologics License Approval
Approval Date: 11 September 2026
Applicant: Scholar Rock, Inc.
BLA: 761463
Intelligence Overview
The U.S. Food and Drug Administration (USFDA) has approved Isembyld® (apitegromab-mstn) injection, developed by Scholar Rock, Inc., under BLA 761463. The approval was granted under Section 351(a) of the Public Health Service Act.
Isembyld is indicated for the treatment of spinal muscular atrophy (SMA) in adults and pediatric patients 2 years of age and older who are currently receiving an SMN2-targeted treatment. The product is authorized as a 150 mg/3 mL injection single-dose vial.
Regulatory Highlight: FDA also granted a Rare Pediatric Disease Priority Review Voucher (PRV) associated with BLA 761463.
Reference: https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2026/761463Orig1s000ltr.pdf