Scholar Rock announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for apitegromab. This muscle-targeted therapy is designed to improve motor function in patients with spinal muscular atrophy (SMA) who are receiving SMN-targeted treatments.
The BLA submission is supported by positive data from the pivotal Phase 3 SAPPHIRE trial and the Phase 2 TOPAZ trial. Scholar Rock has requested Priority Review for the application, which, if granted, could shorten the FDA's review time.
In parallel, the company remains on track to file a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) in the first quarter of 2025. Scholar Rock also plans to initiate the Phase 2 OPAL trial for SMA patients under two years of age in mid-2025.
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