FDA 以应用缺陷为由拒绝 govorestat 用于治疗经典型半乳糖血症（一种罕见疾病）。 FDA rejects govorestat for treating Classic Galactosemia, a rare disease, citing application deficiencies.
FDA 发布了 govorestat 的完整回复函，这是一种治疗经典半乳糖血症的潜在治疗方法，经典半乳糖血症是一种罕见的遗传病，影响了美国约 3,300 名患者。 The FDA has issued a Complete Response Letter for govorestat, a potential treatment for Classic Galactosemia, a rare genetic disease affecting about 3,300 patients in the USA. 该药物在临床试验中表现出希望,但在应用方面却面临着缺陷。 The drug showed promise in clinical trials but faced deficiencies in its application. 该药物的开发者应用疗法计划审查反馈,并寻求举行一次会议,讨论下一步措施,包括可能重新提交或上诉。 Applied Therapeutics, the drug's developer, plans to review the feedback and seek a meeting to discuss next steps, including potential resubmission or appeal. 公司的股票在FDA的决定之后大幅下跌. The company’s stock dropped significantly following the FDA’s decision.