FDA Approves Wakix for Children with Narcolepsy and Cataplexy

Harmony Biosciences has secured a new approval from the U.S. FDA for its drug Wakix. The medication can now be prescribed to children aged six and above who suffer from narcolepsy with cataplexy. This latest decision expands the drug's existing use in adult patients with similar conditions.

Wakix, known chemically as pitolisant, first received FDA approval in 2019. At that time, it was cleared for treating excessive daytime sleepiness in adults with narcolepsy. A year later, in 2020, its label was updated to include adults experiencing cataplexy—a sudden loss of muscle tone often linked to narcolepsy.

The newest approval now extends Wakix's use to pediatric patients. Children as young as six can be prescribed the drug if they have narcolepsy accompanied by cataplexy. Beyond this latest expansion, the company continues to explore further applications for Wakix. Clinical trials and regulatory submissions are underway for other sleep-wake disorders, including narcolepsy type 2 and idiopathic hypersomnia.

Several of these programs are currently under review by both the FDA and the EMA. While the exact number of ongoing regulatory assessments remains undisclosed, the company is actively pursuing additional age groups and indications for the drug.

The expanded label allows Wakix to reach a younger patient group for the first time. Doctors treating children with narcolepsy and cataplexy now have an approved pharmaceutical option. Meanwhile, further research and regulatory reviews could widen the drug's potential applications in the future.