FDA broadens label for Avadel's narcolepsy drug to include children

Avadel Pharmaceuticals has received approval for its once-nightly narcolepsy drug, Lumryz, to be administered to children older than 7 years. The supplemental new drug application (NDA) approval was confirmed on Thursday, enabling Lumryz to treat cataplexy or excessive daytime sleepiness in children with narcolepsy. This approval came after a delay from the FDA, which missed its original decision date of September 7.

Lumryz is now the only FDA-approved treatment that can be taken once nightly for this condition in the pediatric population. Jennifer Gudeman, Avadel’s senior vice president of medical and clinical affairs, emphasized the significance of expanding this treatment to children. According to Avadel, children represent approximately 5% of all narcolepsy patients treated with oxybate. Oxybate, a common prescription for narcolepsy, is also marketed under the name Xyrem by Jazz Pharmaceuticals.

Avadel initially sought FDA approval for Lumryz in November, following its approval for adult use in May 2023. Earlier in 2023, Avadel triumphed in a prolonged legal battle with Jazz Pharmaceuticals over the patent for Xyrem, which requires twice-nightly dosing.

Gudeman highlighted the difficulties faced by patients who use the twice-nightly oxybate products, noting that many find it challenging to maintain the dosing schedule during the night. This disruption can affect the entire household, indicating a significant unmet need for a more convenient dosing option, particularly in the pediatric segment.

The approval of Lumryz for adults was based on positive Phase 3 results from the REST-ON study. This study demonstrated significant and clinically meaningful improvements compared to placebo across all three co-primary endpoints: the maintenance of wakefulness test, the Clinical Global Impression-Improvement, and the mean weekly cataplexy attacks for the three doses evaluated (6 g, 7.5 g, and 9 g).

Lumryz is distributed through a risk evaluation and mitigation strategy (REMS) program and carries a boxed warning. Common adverse effects reported include nausea, dizziness, bed-wetting, headache, and vomiting.

In the previous year, Lumryz generated approximately $28 million in sales. Analysts at investment bank Leerink Partners predicted in August that Lumryz could capture over 20% of the narcolepsy market share. They also noted that while Avadel had succeeded in one patent dispute with Jazz, a court ruled that Avadel could not seek approval for Lumryz for idiopathic hypersomnia until another Jazz patent expires in 2036.