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Scholar Rock aims FDA approval for apitegromab as first muscle-targeted SMA therapy by Sept 2026

Market News
07 Sep 2026
Seeking Alpha
View Source
Neutral
Scholar Rock aims FDA approval for apitegromab as first muscle-targeted SMA therapy by Sept 2026

Scholar Rock is preparing to launch apitegromab, the first therapy targeting muscles for spinal muscular atrophy (SMA), pending FDA approval expected by September 30, 2026. Manufacturing delays have been due to production issues, not drug safety or efficacy, with alternative facilities reducing but not eliminating approval risks. Clinical trial data from the SAPPHIRE study show apitegromab improves motor function significantly in nonambulatory SMA patients when added to existing treatments. The company's valuation heavily depends on this drug's approval and commercial success, with risks tied to regulatory and market execution.

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