FDA Delays Approval of Scholar Rock’s Apitegromab for Spinal Muscular Atrophy
The Food and Drug Administration has rejected the biologics license application (BLA) for apitegromab, a potential muscle-targeted treatment for spinal muscular atrophy (SMA), halting its path to becoming the first therapy of its kind for the debilitating genetic condition. The decision stems from concerns regarding manufacturing processes at Catalent Indiana, a contract manufacturing organization.
This setback impacts individuals living with SMA, a disease characterized by muscle weakness and atrophy, and their families who have anticipated a new treatment option focused on directly strengthening muscles. Scholar Rock, the developer of apitegromab, intends to resubmit the BLA as soon as Catalent Indiana resolves the FDA’s identified issues. The agency’s decision does not question the drug’s safety or efficacy data, but rather focuses on manufacturing quality control.
“We are disappointed with the FDA’s decision, but we are committed to resubmitting the apitegromab BLA as soon as possible,” stated David with L. fish,chairman and CEO of Scholar Rock,in a company release. “We remain focused on working hand-in-hand with the FDA to pursue approval of the first and only muscle-targeted treatment for people living with [spinal muscular atrophy].”
Scholar Rock anticipates resubmitting the application once Catalent Indiana addresses the FDA’s concerns. The company did not provide a specific timeline for resubmission.
