VYKAT XR (diazoxide choline), the first FDA-approved medication specifically indicated for hyperphagia in patients ages 4 and older with Prader-Willi syndrome, is facing increased safety scrutiny following reports of seven deaths and more than 100 serious adverse events.
The reported events included edema and respiratory and cardiac complications. The FDA’s Adverse Event Reporting System has logged 654 total cases tied to the drug, including 112 serious cases. Reports rose sharply from 119 in 2025 to 535 so far in 2026.
PWSA | USA, the Foundation for Prader-Willi Research and the International Prader-Willi Syndrome Organisation issued a joint statement Aug. 11 alerting clinicians and families to the reported adverse events. The statement was developed with input from clinicians experienced in treating Prader-Willi syndrome.
The groups emphasized that the reports do not establish a causal link between VYKAT XR and the outcomes, noting many of the affected patients had complex medical conditions, were taking multiple medications or had severe obesity. No deaths were reported in the drug’s pivotal trial, though an elevated risk of mild swelling was observed.
Rather than calling for the drug’s removal from the market, the groups urged informed prescribing, careful patient selection and close monitoring, particularly for patients with known risk factors such as severe obesity, cardiac disease, pre-existing edema, untreated sleep apnea and significant respiratory compromise.
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