由于制造问题,而不是安全或功效问题,林业发展局拒绝SMA的Apitegromab。 FDA rejects apitegromab for SMA due to manufacturing issues, not safety or efficacy.

美国食品和药物管理局发布了一封完整的回应信,拒绝了Scholar Rock的申请,该申请是针对脊髓肌缩的治疗方法,理由是第三方设施Catalent Indiana的制造问题. The FDA issued a Complete Response Letter rejecting Scholar Rock’s application for apitegromab, a treatment for spinal muscular atrophy, citing manufacturing issues at Catalent Indiana, a third-party facility. 根据检查结果作出的决定并不质疑药物的安全性或效力。 The decision, based on inspection findings, does not question the drug’s safety or efficacy. 学者Rock计划在设施处理这些关切后重新提出申请。 Scholar Rock plans to resubmit its application after the facility addresses the concerns. 该公司仍然致力于为病人提供治疗,其股票在市场前交易中下降了12%以上。 The company remains committed to bringing the treatment to patients, and its shares dropped over 12% in pre-market trading.