Physicians alert clinicians to deaths and serious side effects in users of Neurocrine's new PWS drug
A coalition of doctors treating Prader-Willi syndrome warned that seven patients on Vykat XR have died and over 100 serious adverse events have been reported to the FDA.
Doctors and experts in Prader-Willi syndrome issued a statement to alert clinicians that seven patients taking Vykat XR have died since the drug's FDA approval in March 2025. In addition, the FDA’s Adverse Event Monitoring System has received over 100 reports of serious adverse events, most involving hospital admissions for swelling, breathing problems, or heart complications. The physicians clarified that neither the fatalities nor the severe side effects have been definitively attributed to Vykat XR.
Their purpose is to raise caution among providers when starting the medication for children and adults with the disorder. The notice does not claim a proven causal relationship but urges vigilance. The FDA continues to monitor the situation through its reporting system.
Why it matters
Patients with Prader-Willi syndrome and their doctors need to be aware of possible serious risks linked to a newly approved treatment.
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