FDA approves sodium oxybate to treat cataplexy, EDS in pediatric narcolepsy patients
The approved indication is for patients aged 7 years and up with narcolepsy to treat cataplexy or excessive daytime sleepiness.
FDA approves sodium oxybate to treat cataplexy, EDS in pediatric narcolepsy patients | Image Credit: © Calin - © Calin - stock.adobe.com.
The FDA has approved sodium oxybate (Lumryz; Avadel Pharmaceuticals) extended-release oral suspension to treat cataplexy or excessive daytime sleepiness (EDS) in patients aged 7 years or older with narcolepsy, according to a press release from Avadel.
The decision makes sodium oxybate the only FDA-approved once-at-bedtime oxybate treatment for cataplexy or EDS in among this patient population. Along with the approval, sodium oxybate has been granted Orphan Drug Exclusivity in pediatric narcolepsy patients aged 7 years and older through October 16, 2031.
"I have been prescribing sodium oxybate for children and adolescents with narcolepsy for years as I have seen how effective this medication is and can safely be used," said Anne Marie Morse, DO, pediatric neurologist; sleep medicine specialist, Geisinger Health System, in a statement. "I have also had many families turn down the medication, or discontinue after starting, because of the challenge experienced or feared to experience with a forced awakening causing a purposeful nightly disruption, many times met with an exhausting fight, to take the second dose of first-generation oxybates. I can now offer more options to more patients which allows me to continue my role as a partner in my patients' journeys."
Narcolepsy affects approximately 1 in 2,000 people in the United States, where approximately 5% of patients are younger than 18 years of age. Cataplexy, disrupted nighttime sleep, sleep paralysis, and hallucinations when falling asleep or waking up are symptoms narcolepsy.
The once-at-bedtime formulation extended release sodium oxybate was approved on May 1, 2023 to treat adults with narcolepsy.
According to Avadel, sodium oxybate has a boxed warning as a central nervous system depressant, and for its potential for abuse and misuse, and that the formulation is only available through a restricted program under a Risk Evaluation and Mitigation Strategy called "Lumryz Rems."
"Most common adverse reactions (incidence > 5% and greater than placebo) reported for all doses of LUMRYZ combined in a trial of adults with narcolepsy were nausea, dizziness, enuresis, headache, and vomiting. Similarly, in a trial of pediatric narcolepsy patients receiving immediate-release sodium oxybate, the most commonly observed adverse reactions (incidence ≥5%) were nausea, enuresis, vomiting, headache, decreased weight, decreased appetite, dizziness, and sleepwalking," stated Avadel.
Reference:
Avadel Pharmaceuticals Announces FDA Approval of LUMRYZ™ (sodium oxybate) Extended-Release Oral Suspension (CIII) for the Treatment of Cataplexy or Excessive Daytime Sleepiness in Patients 7 Years of Age and Older with Narcolepsy. Avadel Pharmaceuticals. Press release. October 17, 2024. Accessed October 17, 2024. https://investors.avadel.com/news-releases/news-release-details/avadel-pharmaceuticals-announces-fda-approval-lumryztm-sodium
