Experts At Prader-Willi Point Up Safety Concerns with Neurocrine’s Medication for Uncommon Diseases
On Tuesday, experts in Prader-Willi Syndrome expressed worries about the safety of Neurocrine’s (NBIX.O) new tablet medication Vykat XR, noting severe side effects that have been documented in individuals with the uncommon genetic condition.
The defining symptom of Prader-Willi syndrome, hyperphagia, or feelings of acute and persistent hunger, is caused by deletions on chromosome 15, which alter how genes turn on and off. Vykat XR was licensed last year to treat this condition.
The International Prader-Willi Syndrome Organization and the Foundation for Prader-Willi Research jointly released a statement stating that since Vykat XR became more accessible, post-marketing surveillance has reported serious adverse events, such as edema, respiratory problems, and cardiac problems.
On Wednesday, Neurocrine’s stock dropped 2.3%. According to the statement, as of July 31, there were seven reports of deaths connected to individuals using the medication in the U.S. Food and Drug Administration’s Adverse Event Monitoring System (AEMS).
The most often reported side effects on AEMS were peripheral edema and the medication’s ineffectiveness. A representative for the U.S. Department of Health and Human Services stated, “The FDA is assessing the available data and will determine if regulatory action is needed.”
The joint statement described the findings as alarming and stated that patients and their families should have access to information about the drug’s safety profile. However, it emphasized that there is no proof linking Vykat XR to the outcomes that have been reported.
BMO analyst Evan Seigerman stated “greater physician caution around prescription and risk of regulatory scrutiny might limit demand and curtail the product rollout in coming quarters”. “In the context of a very critical illness, VYKAT XR has an attractive risk-benefit profile. According to Neurocrine, “during our process to acquire Soleno, we conducted extensive diligence on the safety profile, including adverse event data.”
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It noted that 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.”
Neurocrine acquired access to Vykat XR earlier this year when it paid $2.9 billion to acquire Soleno.