Avadel Pharmaceuticals plc a biopharmaceutical company focused on transforming medicines to transform lives, announced today that the U.S. Food and Drug Administration (FDA) has approved its supplemental new drug application (sNDA) for LUMRYZ for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy.

“This approval represents an important milestone for the narcolepsy community, specifically for younger narcolepsy patients and their caregivers who face significant challenges associated with waking up in the middle of the night to complete treatment regimens. With this label expansion, pediatric patients 7 years and older living with narcolepsy now have the same option that adult patients with narcolepsy have – to choose a once-nightly treatment option that does not disrupt sleep for a middle of the night dose,” said Greg Divis, Chief Executive Officer of Avadel Pharmaceuticals. “In less than two years, Avadel has made great strides in establishing our commitment to the development of transformative medicines for sleep disorders. This includes successfully launching LUMRYZ for the adult narcolepsy population, initiating a Phase 3 pivotal trial evaluating LUMRYZ for the treatment of idiopathic hypersomnia, and now the expansion of LUMRYZ into the pediatric narcolepsy population.”

"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, a board-certified and fellowship-trained pediatric neurologist and sleep medicine specialist at Geisinger Health System. “And before this, although numerous families have also witnessed the transformation in their children's lives, 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. The expanded FDA approval for LUMRYZ allows me to now share with my patients and their families that there is an FDA-approved treatment that offers a single bedtime dose of medication, provided in a pre-filled packet. I can now offer more options to more patients which allows me to continue my role as a partner in my patients' journeys."

Narcolepsy is a chronic neurological condition that impairs the brain's ability to regulate the sleep-wake cycle. The condition affects approximately 1 in 2,000 people in the United States, where roughly 5% of patients are under the age of 18. Symptoms of narcolepsy are EDS and may also include a sudden loss of muscle tone usually triggered by strong emotion (cataplexy), disrupted nighttime sleep, sleep paralysis and hallucinations when falling asleep or waking up.

LUMRYZ is a once-at-bedtime formulation extended-release sodium oxybate and was first approved by the FDA on May 1, 2023, for the treatment of cataplexy or EDS in adult patients with narcolepsy.

LUMRYZ was approved by the FDA for use in the treatment of cataplexy or EDS in the pediatric narcolepsy population 7 years and older on October 16, 2024, and was granted Orphan Drug Exclusivity through October 16, 2031.

LUMRYZ has a boxed warning as a central nervous system depressant, and for its potential for abuse and misuse. LUMRYZ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the 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.

 

Source: avadel.com

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