Aug 12 (Reuters) – Experts in Prader-Willi Syndrome on Tuesday raised safety concerns over Neurocrine’s drug Vykat XR, citing serious adverse events reported in patients with the rare genetic disorder.
Vykat XR was approved last year to treat hyperphagia or feelings of intense and persistent hunger, the hallmark symptom of Prader-Willi syndrome, a genetic disorder caused by deletions on chromosome 15 that affect gene expression, or how genes turn on and off.
In a joint statement, Foundation for Prader-Willi Research and the International Prader-Willi Syndrome Organisation said serious adverse events – including edema, respiratory complications and cardiac complications – had been reported through post-marketing surveillance since Vykat XR became more widely available.
Neurocrine shares fell 3.1% in premarket trading on Wednesday.
According to the statement, the U.S. Food and Drug Administration’s Adverse Event Monitoring System (AEMS) showed seven reports of death linked to patients taking the drug as of July 31, 2026.
The company and the U.S. Food and Drug Administration did not immediately respond to a Reuters request for comment.
The organizations said the reports were concerning and that patients and families deserved transparency regarding the drug’s safety profile.
They stressed, however, that the reports do not establish a causal relationship between Vykat XR and the reported outcomes.
“The purpose of this new statement is not to discourage the use of VYKAT XR. Rather, it is to encourage informed prescribing, careful patient selection, and close monitoring, particularly for individuals with known risk factors,” the statement said.
Stat News was the first to report on the matter.
(Reporting by Puyaan Singh in Bengaluru; Editing by Vijay Kishore)
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