The treatment was previously approved for excessive daytime sleepiness in pediatric patients 6 years of age and older with narcolepsy.
Key takeaways:
- The FDA approved the sNDA for WAKIX (pitolisant) to treat cataplexy in pediatric patients aged 6 years and older with narcolepsy.
- This approval expands the drug’s indication, which was previously approved for excessive daytime sleepiness in pediatrics in June 2024.
- WAKIX is a selective histamine 3 receptor antagonist/inverse agonist and is not a controlled substance.
- In pediatric trials, the most common adverse reactions were headache and insomnia.
The US Food and Drug Administration (FDA) has approved Harmony Biosciences’ supplemental new drug application (sNDA) for WAKIX (pitolisant) tablets for the treatment of cataplexy in pediatric patients 6 years of age and older with narcolepsy.
With this latest approval, WAKIX becomes the first and only FDA-approved non-scheduled treatment available for both pediatric and adult narcolepsy patients with or without cataplexy.
“We are excited to achieve this important milestone for pediatric narcolepsy patients who experience cataplexy,” says Kumar Budur, MD, MS, chief medical and scientific officer of Harmony Biosciences, in a release. “With this approval, clinicians now have the option to prescribe WAKIX to treat excessive daytime sleepiness, cataplexy, or both, in patients 6 years and older with narcolepsy.”
The FDA previously approved WAKIX in August 2019 for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy, expanding that approval to include cataplexy in adults in October 2020. In June 2024, the agency approved the drug for the treatment of excessive daytime sleepiness in pediatric patients aged 6 years and older.
“WAKIX is now the first-and-only FDA-approved treatment option for both pediatric and adult narcolepsy patients with or without cataplexy that is not scheduled as a controlled substance, an important distinction that supports its clinical utility,” says Jeffrey M. Dayno, MD, president and chief executive officer of Harmony Biosciences, in a release. “We continue to advance our efforts toward obtaining pediatric exclusivity for WAKIX, which, if granted, would add an additional six months of regulatory exclusivity for this growing franchise.”
WAKIX is a selective histamine 3 (H3) receptor antagonist/inverse agonist. While the precise mechanism of action is unclear, its efficacy could be mediated through its activity at H3 receptors, increasing the synthesis and release of histamine, a wake-promoting neurotransmitter.
In the placebo-controlled phase of the clinical trial conducted in pediatric patients 6 years and older with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and greater than placebo) were headache (19%) and insomnia (7%). According to the manufacturer, the overall adverse reaction profile in the pediatric trial was similar to that seen in the adult clinical trial program.
WAKIX is contraindicated in patients with severe hepatic impairment and in patients with known hypersensitivity to pitolisant. It also prolongs the QT interval and should be avoided in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval.
