FDA Approves Expanded Use of Xyrem to Treat Children with Narcolepsy

October 29, 2018

Rare Daily Staff

The U.S. Food and Drug Administration (FDA) expanded the approved use of Jazz Pharmaceuticals’ Xyrem to include children ages seven and older to treat cataplexy or excessive daytime sleepiness in patients with narcolepsy.

Narcolepsy is a chronic neurologic condition in which the brain is not able to control sleep-wake cycles normally. Patients with narcolepsy have an irresistible urge to sleep during the day. Cataplexy, a major symptom of the disorder in which the muscles weaken when a person feels strong emotions, can cause the head to drop, the face to droop, the jaw to weaken or the knees to give way. Narcolepsy affects an estimated one in 2,000 people in the United States, and symptoms often begin during adolescence. It may take 10 years or more from the time of symptom onset for people with narcolepsy to receive a diagnosis.

“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 FDA approved the use of Xyrem to treat both cataplexy and excessive daytime sleepiness in adult patients with narcolepsy in 2002. The expanded approval for use in children comes after a multisite year-long double-blind phase 2/3 study, which enrolled patients seven to 17 years of age with narcolepsy with cataplexy. The safety profile of Xyrem in children and adolescents in the study were similar to that reported in adults. Results from the study were published in The Lancet Child & Adolescent Health in July 2018, and topline data was presented at the Actionable Patient Safety Solutions meetings in June 2017 and 2018.

October 29, 2018

Photo: Claire Crisp, executive director of Wake Up Narcopepsy

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