FDA approves Wakix for children with narcolepsy and cataplexy

The U.S. Food and Drug Administration (FDA) has expanded the approval of Wakix for younger patients. The medicine, known chemically as pitolisant, can now treat children aged six and older suffering from narcolepsy with cataplexy. This decision follows a successful clinical trial involving 85 children in that age group.

The FDA first approved Wakix in 2019 for adults with narcolepsy. A year later, its use extended to include adults experiencing cataplexy—a sudden loss of muscle tone often triggered by strong emotions.

The latest approval comes after the Phase 3 PEDPLISO study, a randomised, double-blind, placebo-controlled trial. Results showed significant reductions in cataplexy episodes and improvements in excessive daytime sleepiness among the 85 participating children. The study provided the necessary evidence to support the medicine's safety and effectiveness in younger patients.

Harmony Biosciences, the company behind Wakix, announced the label expansion on Tuesday. The firm has also expressed plans to explore further uses for pitolisant, including new formulations and additional medical conditions.

With this approval, Wakix becomes one of the few treatments available for children with narcolepsy and cataplexy. The decision allows doctors to prescribe the medicine to a broader age range, starting from six years old. Harmony Biosciences continues to focus on expanding the medicine's applications in the future.