FDA OKs Harmony Bio New Narcolepsy Drug

FDA has approved a Harmony Biosciences NDA for Wakix (pitolisant) for treating excessive daytime sleepiness in adult patients with narcolepsy. The company says the drug is the first with this indication that is not scheduled as a controlled substance. It says that the selective histamine 3 (H) receptor antagonist/inverse agonist works through a novel mechanism of action to increase the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain.

Approval is based on data from two multicenter, randomized, double-blind, placebo-controlled studies (HARMONY 1 and HARMONY 1bis) that included 261 patients who were randomized to receive Wakix, placebo, or active control. Wakix “demonstrated a statistically significant improvement in EDS as measured by the Epworth Sleepiness Scale (ESS) score,” Harmony says. The most common adverse reactions reported by patients were insomnia (6%), nausea (6%), and anxiety (5%).