FDA Approves Expanded use of Pitolisant for Treatment of Cataplexy in Adults With Narcolepsy

  • cataplexy
  • Excessive daytime sleepiness
  • FDA Approvals
  • Narcolepsy
  • Pitolisant
  • Sleep
  • Sleep disorders

The Food and Drug Administration (FDA) approved pitolisant (Wakix; Harmony Biosciences Plymouth Meeting, PA)—treatment of cataplexy in adults with narcolepsy. Pitolisant is the first and only treatment not categorized as a controlled substance that is approved by the FDA for treating symptoms of narcolepsy.
FDA approval for the expanded indication is based on the results of 2 randomized controlled trials Harmony 1 (NCT01067222) and Harmony CTP (NCT01800045) from the clinical development program for pitolisant. The FDA reviewed a reanalysis of the HARMONY 1 data submitted during the NDA review, and acknowledged that those analyses confirmed a statistically significant reduction in the rate of cataplexy with pitolisant treatment compared with placebo. 

"All people living with narcolepsy have excessive daytime sleepiness and up to two-thirds of them also experience cataplexy, which is one of the most debilitating symptoms of this chronic, rare neurologic disorder," said Harmony's chief medical officer, Jeffrey Dayno, MD. "Today's FDA approval of the cataplexy indication for Wakix, coupled with it being the first and only nonscheduled treatment option approved for adults with narcolepsy to treat both excessive daytime sleepiness or cataplexy, provides an opportunity for Wakix to offer broad clinical utility to healthcare professionals managing adults living with narcolepsy."

In the placebo-controlled clinical trials conducted in participants with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and twice that of placebo) for pitolisant were insomnia (6%), nausea (6%), and anxiety (5%). Other adverse reactions that occurred at ≥2% and more frequently than in participants treated with placebo included headache, upper respiratory infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash.

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

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