Avadel Pharmaceuticals said its once-nightly narcolepsy drug Lumryz can now be used to treat children older than 7 years of age.
The Thursday approval of the supplemental NDA for Lumryz, allowing the drug to treat cataplexy or excessive daytime sleepiness in children with narcolepsy, came after FDA missed its Sept. 7 PDUFA date. Avadel says Lumryz is now the only FDA-approved, once-per-night treatment in this population.
It’s “really important” to expand a once-nightly dose into the pediatric market, according to Jennifer Gudeman, Avadel’s senior VP of medical and clinical affairs. Children currently represent approximately 5% of all oxybate-treated narcolepsy patients, according to the company. Oxybate is commonly prescribed to patients with narcolepsy, and it is also marketed as Xyrem by Jazz Pharmaceuticals.
Avadel filed for approval with the FDA in November following Lumryz’s approval in adults in May 2023. Avadel won a multi-year court battle earlier in 2023 with Jazz over the latter company’s patent for Xyrem, which is dosed twice per night.
“There’s people who have started on the twice-nightly oxybate products, and they just have not been able to sustain being able to dose in the middle of the night,” Gudeman said. “We hear about the fact that it can turn into a disruption to the whole household and so there absolutely was an unmet need in adults. But I think based upon all of the feedback that we received, many believe that unmet need is even greater in the pediatric population because you’re disrupting multiple peoples’ sleep.”
Lumryz was approved in adults after positive Phase 3 results in the REST-ON study showed highly statistically significant (p<0.001) and clinically meaningful improvement compared to placebo across all three co-primary endpoints (a maintenance of wakefulness test, the Clinical Global Impression-Improvement and mean weekly cataplexy attacks) for all three doses evaluated (6 g, 7.5 g and 9 g).
Lumryz is only available through a risk, evaluation and mitigation strategy (REMS) program, and its label comes with a boxed warning. The most common adverse events included nausea, dizziness, enuresis, headache and vomiting.
The drug generated about $28 million in sales last year, and investment bank Leerink Partners analysts in August estimated that Lumryz could grab more than 20% of the market share in narcolepsy. They also pointed out that while Avadel had won one patent fight against Jazz, the court also ruled Avadel couldn’t seek approval for Lumryz in idiopathic hypersomnia until another Jazz patent expires in 2036.
Editor’s note: This story has been updated to clarify that Lumryz is also an oxybate.