STAT+: Concerns grow on safety issues tied to Neurocrine Biosciences rare disease drug — SkimNews
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- Vykat XR, approved to treat Prader-Willi syndrome, has been linked to seven deaths and nearly 100 hospitalizations since gaining approval 18 months ago, according to case reports submitted to regulators.
- STAT obtained the case reports on those deaths and hospitalizations through the Freedom of Information Act, and the article notes 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 two months after releasing an open letter on potential safety issues tied to the therapy.
- Some experts say the available evidence is already sufficient to conclude that Vykat is causing unacceptable harm, despite the lack of proven causality in individual case reports.
- Prader-Willi syndrome, the condition Vykat XR treats, can cause an insatiable feeling of hunger in patients.
Why it matters: Seven deaths and roughly 100 hospitalizations tied to an 18-month-old rare-disease approval puts the FDA and Neurocrine under pressure to act — either through new safety warnings, label changes, or a deeper review of the complex, multi-medication cases that complicate proving Vykat XR's direct role. Patient advocates who flagged these issues publicly are now demanding accountability that could reshape how post-market surveillance functions for ultra-rare-disease drugs.
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