STAT+: Concerns grow on safety issues tied to Neurocrine Biosciences rare disease drug

Two months after the release of an open letter on potential safety issues tied to a rare disease drug sold by Neurocrine Biosciences, patient advocates and some experts are expressing growing alarm over the perceived risks of the therapy. The drug, approved to treat Prader-Willi syndrome, which can cause an insatiable feeling of hunger, has been linked to the deaths of seven people and the hospitalization of nearly 100 others — all since the treatment, called Vykat XR, was approved 18 months ago. Case reports on those deaths and hospitalizations that were submitted to regulators, and that were obtained by STAT through the Freedom of Information Act, do not prove causality; many of the Vykat cases involved complex medical issues and multiple medications. But some experts say the evidence is sufficient to conclude Vykat is causing unacceptable harm. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe