STAT+: Neurocrine Biosciences rare disease drug possibly tied to safety issues, experts say
A newly approved drug for Prader-Willi syndrome, a rare disease that causes an insatiable desire to eat, may be tied to a series of patient deaths, experts say.
A coalition of medical professionals and specialists dealing with Prader-Willi syndrome, a rare condition characterized by an uncontrollable appetite, alerted healthcare providers to a series of fatalities and reports of serious complications linked to Vykat XR, a recently approved medication manufactured by Neurocrine Biosciences. The Food and Drug Administration had granted Vykat approval in March 2025 to manage the excessive hunger experienced by individuals with Prader-Willi syndrome, both children and adults (PWS).
Since its authorization, there have been seven documented instances of death among individuals treated with Vykat. Moreover, over 100 accounts of severe adverse reactions, predominantly comprising patients being hospitalized due to swelling, respiratory, and cardiac issues, have been submitted to the FDA’s adverse event monitoring system.
The group of medical experts and physicians issued this statement to heighten awareness among healthcare providers regarding the potential risks associated with Vykat XR for patients with PWS.
However, it remains unconfirmed whether the fatalities or the severe side effects are directly attributable to the drug. The physicians and experts affirmed that further investigation is warranted to establish a definitive connection between Vykat XR and the reported occurrences. For those interested in a comprehensive account of this matter, they are directed to continue reading STAT+.
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