STAT+: Concerns grow on safety issues tied to Neurocrine Biosciences rare disease drug — SkimNews
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- Vykat XR, approved 18 months ago to treat Prader-Willi syndrome, has been linked to seven deaths and nearly 100 hospitalizations, according to case reports submitted to regulators.
- Neurocrine Biosciences faces mounting scrutiny two months after the release of an open letter flagging potential safety issues tied to the drug.
- STAT obtained the case reports through the Freedom of Information Act; the reports do not prove causality, as many cases involved complex medical issues and multiple medications.
- Patient advocates and some experts are expressing growing alarm, with some saying the evidence is sufficient to conclude Vykat is causing unacceptable harm.
- Prader-Willi syndrome, the condition Vykat XR treats, can cause an insatiable feeling of hunger in patients.
Why it matters: With 7 reported deaths and ~100 hospitalizations tied to a drug on the market for just 18 months, regulators may face pressure to revisit Vykat XR's approval — and Neurocrine Biosciences could see its rare disease franchise and patient trust threatened if the safety signal solidifies.
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