FDA Approves Drug to Treat Excessive Daytime Sleepiness for Narcolepsy Patients


The approval marks the first ever treatment for patients with narcolepsy that is not scheduled as a controlled substance by the DEA.

A new treatment for excessive daytime sleepiness (EDS) in adult patients with narcolepsy may hit the market by the end of the year.

The US Food and Drug Administration (FDA) approved WAKIX (pitolisant) as the first treatment for patients with narcolepsy that is not scheduled as a controlled substance by the US Drug Enforcement Administration (DEA).

The once daily drug is developed by Harmony Biosciences.

"We are extremely proud to bring WAKIX to market for those living with narcolepsy, a chronic, debilitating, rare neurologic disorder," Harmony's Chairman and Chief Executive Officer, John C. Jacobs, said in a statement. "At Harmony, we share a vision to develop novel treatment options for people living with rare diseases, with a focus on those that affect the central nervous system. The approval of WAKIX strengthens our commitment to making that vision a reality."

WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist that works through a mechanism of action to increase the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain.

"The approval of WAKIX provides healthcare professionals managing people living with narcolepsy a new and important treatment option for their patients," Harmony's Chief Medical Officer, Jeffrey Dayno, MD, said in a statement. "Additionally, WAKIX is the only non-scheduled treatment option approved for adult patients with narcolepsy, and it offers an important benefit/risk profile to address the unmet medical need that exists in people living with narcolepsy."

The efficacy of WAKIX for the treatment of EDS in adult patients with narcolepsy was evaluated in 2 multicenter, randomized, double-blind, placebo-controlled studies that included 261 patients that received the drug, placebo, or active control.

Treatment duration was 8 weeks, with a 3-week dose titration phase followed by a 5-week stable dose phase.

In both of these studies, WAKIX demonstrated a statistically significant improvement in EDS. In the placebo-controlled trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (occurring in ≥5% of patients and at twice the rate of placebo) with the use of WAKIX were insomnia (6%), nausea (6%), and anxiety (5%).

WAKIX was first granted orphan drug status by the FDA to treat narcolepsy in 2010.

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