FDA Expands Wakix Approval for Childhood Narcolepsy and Cataplexy Treatment

Harmony Biosciences has secured a new approval for its narcolepsy drug, Wakix. The U.S. Food and Drug Administration (FDA) expanded the medication's label on Tuesday, allowing its use in younger patients. This marks another step in the drug's growing range of approved treatments since its initial launch in 2019.

The FDA first greenlit Wakix in 2019 for adults suffering from excessive daytime sleepiness caused by narcolepsy. A year later, its approved uses widened to include adults experiencing cataplexy—a condition linked to narcolepsy that causes sudden muscle weakness.

This week's decision further broadens access. The updated label now permits Wakix (pitolisant) for children aged six and older with narcolepsy and cataplexy. Previously, the minimum age for treatment in the U.S. had been set at six years for narcolepsy with cataplexy, but the latest expansion solidifies this age threshold for both conditions.

Jeffrey Dayno, CEO of Harmony Biosciences, highlighted the company's plans to continue developing pitolisant. Future efforts will focus on exploring additional medical uses and refining next-generation formulations of the drug.

With this label update, Wakix becomes one of the few medications approved for paediatric narcolepsy and cataplexy in the U.S. The decision provides doctors with more options for treating younger patients. Harmony Biosciences now aims to build on this progress with further research and development.