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.
免责声明：投资有风险，本文并非投资建议，以上内容不应被视为任何金融产品的购买或出售要约、建议或邀请，作者或其他用户的任何相关讨论、评论或帖子也不应被视为此类内容。本文仅供一般参考，不考虑您的个人投资目标、财务状况或需求。TTM对信息的准确性和完整性不承担任何责任或保证，投资者应自行研究并在投资前寻求专业建议。