尽管结果是积极的,但FDA因制造问题而暂停对儿童门克斯病的CUTX-101的审查. FDA halts review of CUTX-101 for pediatric Menkes disease due to manufacturing issues, despite positive results.

美国食品和药物管理局发布了针对儿童门克斯病的治疗方法CUTX-101的完整回应信,理由是生产设施的制造缺陷,但它没有质疑该药物的安全性或有效性. The FDA issued a Complete Response Letter for CUTX-101, a treatment for pediatric Menkes disease, citing manufacturing deficiencies at the production facility, though it did not question the drug’s safety or efficacy. 该决定中止了优先审查,是在2025年9月重新检查之后作出的。 The decision, which halted the Priority Review, follows a September 2025 re-inspection. Sentynl治疗学,现在负责监督开发工作,计划解决这些问题并重新提出申请。 Sentynl Therapeutics, now overseeing development, plans to address the issues and resubmit the application. 该药物显示出积极的临床结果,改善了早期治疗患者的生存。 The drug showed positive clinical results, improving survival in early-treated patients. 如果获得批准,Sentynl公司将向Cyprium治疗系统转拨一个儿科疾病优先审查券,后者也有资格获得特许使用费和最高1.29亿美元的里程碑付款。 If approved, Sentynl will transfer a Rare Pediatric Disease Priority Review Voucher to Cyprium Therapeutics, which is also eligible for royalties and up to $129 million in milestone payments.