FDA has approved Scholar Rock’s spinal muscular atrophy treatment apitegromab, branded as Isembyld, following delays tied to manufacturing issues at a troubled factory. The injectable is positioned for patients on standard of-care therapies, with trial results showing motor-function improvements. For SMA development programs, the approval also underscores the manufacturing execution hurdle that can delay late-stage timelines even when clinical efficacy is already established. Scholar Rock will now transition from label execution to scaling and post-approval evidence generation. The FDA decision places additional scrutiny on ongoing quality and supply stability as the company works to expand manufacturing capacity and ensure uninterrupted patient access.
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