Scholar Rock Holding (SRRK) said Wednesday it has submitted a Biologics License Application to the US Food and Drug Administration for apitegromab, an experimental therapy for spinal muscular atrophy, or SMA.
The company said the submission is based on data from a phase 3 trial showing that patients receiving apitegromab achieved a "statistically significant" 1.8-point improvement in motor function over placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at 52 weeks.
The FDA previously granted apitegromab Fast Track, Orphan Drug, and Rare Pediatric Disease designations in SMA, Scholar Rock said.
The company said it has requested Priority Review, which, if granted, would shorten the FDA's review period to six months from the date of filing acceptance.
Scholar Rock said it plans to submit a Marketing Authorization Application to the European Medicines Agency in Q1.
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