Ireland-based Jazz Pharmaceuticals is celebrating a regulatory win for its narcolepsy drug Xyrem (sodium oxybate), which is the first medicine approved to treat cataplexy or excessive daytime sleepiness in pediatric patients with the disorder. The drug was originally approved by the FDA in 2002, however Jazz acquired the drug as part of its buyout of Orphan Medical in 2005.
“Narcolepsy is often misunderstood, misrepresented, misdiagnosed and underdiagnosed, especially in children,” said Claire Crisp, executive director of Wake Up Narcolepsy and mother of a child with narcolepsy. “This approval of Xyrem in pediatric patients is a significant step forward for the narcolepsy community as we work to elevate awareness of the condition in children and ensure patients, both pediatric and adult, have meaningful treatment options available.”
The approval of Xyrem in patients aged seven and older could help those with narcolepsy access treatment sooner, as the condition usually begins in adolescence. About one in every 2,000 individuals in the US is believed to be affected by narcolepsy, however about half of them might not know it due in part to the difficulty in diagnosing the condition.
Excessive daytime sleepiness is one of the major symptoms of narcolepsy, in which patients are unable to control the urge to sleep during the day. Muscle weakness, known as cataplexy, also contributes to the disabling nature of narcolepsy as patients may experience weak knees, loss of head and neck control and facial drooping.
“Xyrem is the only FDA-approved treatment available for excessive daytime sleepiness and cataplexy in narcolepsy for adult patients,” said Dr. Jed Black, senior vice president, Sleep and CNS Medicine at Jazz Pharmaceuticals and adjunct professor, Stanford Center for Sleep Sciences and Medicine. “In a pivotal study we demonstrated both safety and efficacy of Xyrem in pediatric patients with narcolepsy. We are pleased to lead the sleep community in advancing the science and identifying meaningful treatment options for children and adolescents.”
Results from the Phase II/III EXPRESS clinical trial involving pediatric narcolepsy patients were used to support Jazz Pharmaceuticals’ supplemental new drug application (sNDA) to the FDA. Patients included those who had, and had not, previously taken Xyrem, with some patients being randomly assigned to receive a placebo after being established on a stable dose of the drug. Those patients switched from Xyrem to placebo experienced about 13 more narcolepsy attacks each week, compared with patients who continued to receive the active treatment.
However, two serious adverse events were reported in which one patient suffered acute psychosis and another experienced suicidal ideation. Despite this, Jazz reported that the safety profile was similar to that seen in previous studies of adult patients. The company also has a Risk Evaluation and Mitigation Strategy (REMS) program in place for Xyrem with the goal of informing doctors and patients about the other risks of taking the drug, which include CNS and respiratory depression.
Regardless of the risks of the drug, the approval will certainly widen the eligible patient population for Xyrem, which brought in $1.2 billion in net sales in 2017. This number represented a seven percent increase over 2016, with sales likely to increase even more this year.
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