By Puyaan Singh
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 2.7% on Wednesday.
According to the joint 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.
Peripheral edema and the drug being ineffective were the highest reported reactions on FDA’s AEMS.
The joint statement called the reports concerning and said patients and families deserved transparency on the drug’s safety profile, but stressed they do not establish a causal link between Vykat XR and the reported outcomes.
Even so, “increased physician caution around prescribing and risk of regulatory scrutiny could limit uptake and constrain the product launch in coming quarters,” said BMO analyst Evan Seigerman.
“VYKAT XR has a compelling risk-benefit profile in the context of a very serious disease. Neurocrine conducted extensive diligence on the safety profile, including adverse event data, during our process to acquire Soleno,” said Neurocrine.
It added it is “engaged with the FDA, patient advocacy communities and prescribers to continue to assess all available data from postmarketing surveillance as the prescribing population expands.”
Earlier this year, Neurocrine bought Soleno for $2.9 billion, gaining access to Vykat XR.
Stat News was the first to report on the matter.
(Reporting by Puyaan Singh in Bengaluru, additional reporting by Sriparna Roy; Editing by Vijay Kishore)
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