STAT+: Neurocrine Biosciences rare disease drug possibly tied to safety issues, experts say — SkimNews
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- Physicians and Prader-Willi syndrome experts notified clinicians on Tuesday of patient deaths and severe side effects potentially associated with Vykat XR, a newly approved Neurocrine Biosciences 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 (PWS).
- Seven people prescribed Vykat XR 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 reported to the FDA since the drug's clearance.
- The experts' statement said the intention is to increase awareness of risks for PWS patients starting Vykat XR, while noting that neither the deaths nor the severe side effects have been definitively linked to the drug.
Why it matters: Seven deaths and over 100 serious adverse events tied to a drug approved just months ago put early safety pressure on Neurocrine Biosciences and the FDA's March 2025 decision. Because no causal link has been confirmed, clinicians and regulators face an ambiguous signal: the data could prompt label changes, prescribing caution, or deeper post-market review for the only approved PWS hyperphagia treatment.
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