Concerns grow on safety issues tied to Neurocrine Biosciences rare disease drug By Adam FeuersteinOct. 13, 2026

Concerns grow on safety issues tied to Neurocrine Biosciences rare disease drug Former FDA regulator wants Prader-Willi therapy withdrawn; others urge caution By Adam FeuersteinOct. 13, 2026 Adam Feuerstein, a senior writer and biotech columnist, is the author of Adam’s Biotech Scorecard, a subscriber-only newsletter about the crossroads of drug development, business, Wall Street, and biotechnology. https://www.statnews.com/2026/10/13/neurocrine-biosciences-prader-willi-vykat-xr/?utm_campaign=the_readout&utm_medium=email&_hsenc=p2ANqtz-8jTSF7KQ1LMyO8XpnkZigFnrRTFPsNVQjnEfmQ0ALOTl2mloSjvB8DCXbcsX48J9vk1NzVXE6QSBqABR7XMpI_vZVc3Q&_hsmi=443884717&utm_content=443884717&utm_source=hs_email Concerns grow over Neurocrine's Prader-Willi drug after patient deaths Neurocrine Biosciences is under mounting scrutiny over its drug, Vykat XR — the first FDA-approved treatment for the insatiable hunger associated with the rare disease called Prader-Willi syndrome. There have been reports of seven deaths associated with the drug, STAT’s Adam Feuerstein writes, and nearly 100 hospitalizations since its approval 18 months ago. But others argue that the drug is desperately needed and that there is not definitive proof that the treatment caused the deaths. Among those ringing the alarm is Karen Vogt, a pediatric endocrinologist and former medical officer at the Food and Drug Administration whose teenage son has Prader-Willi. “In my opinion, all of these deaths are related to Vykat,” Vogt said. “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.” Neurocrine Biosciences maintains that Vykat represents an important advance for patients and plans to launch an additional safety study early next year.