Matthew Herper· @matthewherper · X·· 4 小时前精选AI 评分78
AI 导读
STAT记者Adam Feuerstein报道,Neurocrine的罕见病药物Vykat获批用于Prader-Willi综合征18个月后,已关联7例死亡和100例住院。记者通过《信息自由法》获得这7例死亡的病例报告,报告未证明因果关系,但部分专家认为证据足以认定Vykat造成不可接受的伤害。儿科内分泌学家、FDA前医疗官员Karen Vogt表示,7例死亡均与Vykat相关,其中6例死于心脏或呼吸衰竭,1例死于严重脑损伤,均由液体潴留或高血糖诱发,她认为FDA应将该药撤市。
推荐理由
通过信息公开申请获得的7例死亡病例报告,呈现Vykat获批后安全性争议的具体证据与专家分歧。
正文
Must read. A worrisome story well told.
Adam at his best.
New story from me: Concerns grow on safety issues tied to Neurocrine $NBIX rare disease drug, Vykat. 18 months after its approval to treat patients with Prader-Willi syndrome, Vykat has been tied to seven deaths and 100 hospitalizations. I obtained case reports on the seven deaths via the Freedom of Information Act. The reports do not prove causality, but some experts say the evidence is sufficient to conclude Vykat is causing unacceptable harm. “In my opinion, all of these deaths are related to Vykat,” said Karen Vogt, a pediatric endocrinologist and former medical officer at the Food and Drug Administration whose teenage son has Prader-Willi. “Six of the seven patients died from heart or respiratory failure, the seventh died from a severe brain injury. All of the deaths were precipitated by significant fluid retention or hyperglycemia that are known and concerning side effects of Vykat,” she added. Vogt believes the FDA should withdraw Vykat from the market. It should never have been approved, she told me. Story here: https://www.statnews.com/2026/10/13/neurocrine-biosciences-prader-willi-vykat-xr/在 X 查看被引用的帖子
来源:Matthew Herper · x.com