Scholar Rock secures first FDA approval for muscle-targeting rare disease drug

Scholar Rock's apitegromab, branded as Isembyld, received FDA approval as the first muscle-targeted treatment to improve motor function in spinal muscular atrophy (SMA) patients aged two and older undergoing SMN2-targeted therapy, following a challenging regulatory path including a prior rejection due to manufacturing compliance issues.

This Wire brief sits within Fusion42's coverage of Biotech, and 9 sources have reported it between 12 Sep 2026 and 14 Sep 2026.