FDA expands narcolepsy drug Wakix to treat children as young as six

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 move follows years of gradual regulatory progress for the treatment.

Wakix, known generically as pitolisant, first entered the market in Europe around 2016. It was later approved in the U.S. in 2019 for treating excessive daytime sleepiness in adults with narcolepsy. The following year, its label grew to include adults suffering from cataplexy—a sudden loss of muscle tone often linked to narcolepsy.

This week's FDA decision extends Wakix's reach further. The drug can now be prescribed to children aged six and older who experience narcolepsy with or without cataplexy. Outside the U.S., Wakix has already been cleared for similar use in adults, adolescents, and children from six years old, as recognised by Swissmedic's Orphan Drug designation.

CEO Jeffrey Dayno highlighted the company's broader ambitions for the drug. Plans include exploring additional medical uses and developing next-generation formulations to increase its value.

The latest approval widens Wakix's patient base in the U.S. to include younger children with narcolepsy. The company continues to pursue further expansions, aiming to strengthen the drug's role in treating sleep disorders. Regulatory milestones in Europe and the U.S. mark its steady growth since 2016.