The Food and Drug Administration (FDA) has approved Wakix® (pitolisant; Harmony Biosciences) for the treatment of cataplexy in adults with narcolepsy.

Wakix is a first-in-class, selective histamine-3 receptor antagonist/inverse agonist that works by increasing the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain. It is the first treatment for narcolepsy that is not scheduled as a controlled substance.

The approval was based on data from 2 multicenter, randomized, double-blind, placebo-controlled phase 3 trials (HARMONY CTP and HARMONY 1) that evaluated the efficacy and safety of pitolisant for the treatment of cataplexy in patients aged 18 years and older with narcolepsy. HARMONY CTP included 105 patients who had at least 3 cataplexy attacks per week and an Epworth Sleepiness Scale (ESS) score of at least 12. HARMONY 1 included a subset of 49 patients with a history of cataplexy and an ESS score of at least 14. 

Findings from HARMONY CTP showed that compared with baseline, the weekly rate of cataplexy decreased over the 4-week stable dosing period from 9.1 (95% CI, 7.6-11.0) to 2.3 (95% CI, 1.5-3.4) with pitolisant and from 7.3 (95% CI, 6.0-8.9) to 4.5 (95% CI, 2.9-7.0) with placebo (rate ratio 0.51 [95% CI, 0.44-0.60]). Additionally, in HARMONY 1, pitolisant demonstrated a statistically significantly greater improvement in the change from baseline in geometric mean daily rate of cataplexy at week 8 compared with placebo.

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Wakix is already indicated for the treatment of excessive daytime sleepiness in adults with narcolepsy. The product is available as 4.45mg and 17.8mg tablets.

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  1. Harmony Biosciences receives FDA approval for expanded use of Wakix® (pitolisant) for the treatment of cataplexy in adult patients with narcolepsy. [press release]. Plymouth Meeting, PA: Harmony Biosciences, LLC; October 14, 2020. 
  2. Wakix
  3. ® [package insert]. Plymouth Meeting, PA: Harmony Biosciences, LLC; 2020.