15 October 2020 - - The US Food and Drug Administration has approved WAKIX (pitolisant) for the treatment of cataplexy in adult patients with narcolepsy, US-based pharmaceutical company Harmony Biosciences Holdings, Inc. (NASDAQ: HRMY) said.

WAKIX is the first and only treatment approved by the FDA for people with excessive daytime sleepiness or cataplexy associated with narcolepsy that is not scheduled as a controlled substance by the US Drug Enforcement Administration.

WAKIX received FDA approval for the treatment of excessive daytime sleepiness in adult patients with narcolepsy in August 2019.

WAKIX, a first-in-class medication, is a selective histamine 3 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. WAKIX is administered orally, once daily in the morning upon wakening.

FDA approval of WAKIX for the treatment of cataplexy in adult patients with narcolepsy is based on the results from two randomized, controlled trials (HARMONY CTP and HARMONY 1) from the clinical development program for WAKIX.

After a complete response letter for the cataplexy indication was issued by the FDA in August 2019, Harmony met with the Agency in December 2019 to discuss the deficiencies cited in the CRL. 

After that meeting, the FDA agreed to review the reanalysis of the HARMONY 1 data that were submitted during the NDA review, after which the Agency acknowledged that those analyses confirmed that a statistically significant reduction in the rate of cataplexy for WAKIX compared to placebo was demonstrated, which supported the positive results from the HARMONY CTP trial. 

Subsequently, the FDA recommended that Harmony submit a resubmission to the CRL, which Harmony submitted in August 2020 and has led to this FDA approval of the cataplexy indication for WAKIX.

Cataplexy is characterized by sudden, temporary loss of muscle tone and is often triggered by strong emotions, such as excitement or laughter. Cataplexy can be subtle, such as drooping of eyelids, or severe, such as knee buckling or total body collapse.