FDA approves Wakix for children with narcolepsy and cataplexy

The U.S. Food and Drug Administration (FDA) has expanded the approval of Wakix for children with narcolepsy. The drug, known generically as pitolisant, can now be prescribed to patients aged six and older who suffer from cataplexy. This decision follows earlier approvals for adults with similar conditions since 2019.

Wakix first gained FDA approval in 2019 for treating excessive daytime sleepiness in adults with narcolepsy. The following year, its label was updated to include adults experiencing cataplexy—a sudden loss of muscle tone often triggered by strong emotions.

The latest expansion covers pediatric patients aged six and above. This approval was based on results from the Phase 3 HARMONY III clinical trial, which showed a significant drop in cataplexy attacks and improved daytime alertness in children. The study provided the necessary evidence for the FDA to extend the drug's use to younger patients.

Harmony Biosciences, the company behind Wakix, continues to explore broader applications for pitolisant. Plans include developing next-generation formulations and pursuing additional indications beyond narcolepsy and cataplexy.

With this label expansion, Wakix becomes one of the few treatments available for children with narcolepsy and cataplexy. The drug's approval now spans both adult and pediatric populations, offering a wider range of patients access to this therapy. Harmony Biosciences remains focused on further research to expand its use.