Scholar Rock announced that positive study results from its pivotal Phase 3 SAPPHIRE trial for apitegromab were published in the peer-reviewed journal The Lancet Neurology. The publication reinforces that children and adults with spinal muscular atrophy (SMA) showed improved motor function with apitegromab.
The data highlighted that patients receiving apitegromab demonstrated significant motor function gains, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE), while the placebo group experienced decreased motor function. This occurred despite all participants receiving ongoing survival motor neuron (SMN)-targeted treatment.
The publication further confirmed that apitegromab was well-tolerated across all age groups, with no new safety findings observed. The Biologics License Application (BLA) for apitegromab is currently under FDA priority review with a PDUFA date of September 22, 2025, and the Marketing Authorisation Application (MAA) is under review by the European Medicines Agency (EMA).
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