STAT+: Neurocrine Biosciences rare disease drug possibly tied to safety issues, experts say
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- Physicians and experts in Prader-Willi syndrome notified clinicians on Tuesday about patient deaths and severe side effects potentially associated with Vykat XR, in a statement aimed at raising awareness of risks for PWS patients starting the drug.
- Vykat XR was approved by the FDA in March 2025 to curb the intense hunger sensation in children and adults with Prader-Willi syndrome.
- Seven people prescribed the drug have died, according to the FDA's Adverse Event Monitoring System.
- More than 100 reports of serious adverse events — mostly hospitalizations for swelling, respiratory, and heart complications — have been filed with the FDA since approval.
- The expert group emphasized that neither the deaths nor the severe side effects have been definitively linked to the drug.
Why it matters: For the small Prader-Willi syndrome community with no other approved hyperphagia therapy, seven deaths and 100+ serious adverse events logged since Vykat XR's March 2025 clearance force an early safety reckoning — though the experts' explicit caveat that no causal link has been proven means regulators and Neurocrine Biosciences must decide whether to escalate warnings now or await formal investigation.
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