Prader-Willi experts flag safety risks linked to Neurocrine’s rare disease drug

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)