Two months after an open letter raised safety concerns about Neurocrine Biosciences' rare disease drug Vykat XR, patient advocates and some experts are growing increasingly alarmed. The therapy, approved for Prader-Willi syndrome, has been tied to the deaths of seven people and the hospitalization of nearly 100 others since it reached the market 18 months ago, according to case reports obtained by STAT through a Freedom of Information Act request.
Those reports do not prove the drug caused the harms; many cases involved complex medical issues and multiple medications. But the divide among experts is sharp. A former FDA regulator has called for the drug to be withdrawn, while others argue for caution rather than immediate removal. The disagreement leaves regulators and clinicians weighing uncertain evidence against the needs of a vulnerable patient population.