STAT记者Adam Feuerstein通过《信息自由法》获取的病例报告显示,Neurocrine的罕见病药物Vykat获批用于治疗Prader-Willi综合征18个月后,已关联7例死亡和100例住院。
通过信息自由法案获取的7例死亡病例报告,呈现Vykat获批18个月后的安全性争议与专家分歧。
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/
来源:Adam Feuerstein ✡️ · x.com