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The Food and Drug Administration late Friday approved Harmony Biosciences' application to expand the use of its flagship sleep disorder drug Wakix.

The Plymouth Meeting biopharmaceutical company now has clearance to market the narcolepsy drug to pediatric patients ages 6 and older.

Wakix initially received marketing clearance from the FDA in 2019 as a treatment for excessive daytime sleepiness in adult patients with narcolepsy. The following year the company received expanded approval to also market the drug to narcolepsy patients with cataplexy.

Narcolepsy, a sleep disorder that causes overwhelming daytime drowsiness, afflicts an estimated 125,000 to 200,000 people in the U.S. That figure includes about 4,000 pediatric patients with the condition, according to Harmony (NASDAQ: HRMY). Cataplexy is characterized by the sudden loss of muscle tone and control while a person is awake. The condition is often triggered by sudden, strong emotions such as laughter, fear, anger, stress or excitement.

Dr. Kumar Budur, the chief medical and scientific officer at Harmony, said the company intends to talk with the FDA about a "path forward" for getting the drug approved for a cataplexy indication in pediatric narcolepsy patients.

The market for narcolepsy drugs in the U.S. could top $6 billion by 2030, according to data from research firm ResearchAndMarkets.com.

Harmony's stock was trading up more than 4% at $31.36 per share in mid-morning trading Monday.

"We are very pleased with the Agency's timely review and approval of Wakix for pediatric narcolepsy patients with excessive daytime sleepiness," said Dr. Jeffrey M. Dayno, CEO of Harmony, in a statement released Monday morning.

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HARMONY BIOSCIENCES

The FDA ruling also fulfills one of the two requirements needed for pediatric exclusivity, which would extend Harmony's patent protection on Wakix by another six months to September of 2030 — a significant revenue opportunity for a drug Harmony expects to produce sales of between $700 million and $720 million this year. The other requirement relates to clinical study data submissions.

During the first quarter of 2024, Wakix generated sales of $154.6 million, up 30% from the same period the previous year. In 2023, Wakix produced revenue of $582 million for Harmony — a 33% increase over sales in 2022.

Harmony's next step is filing a supplemental new drug application for pitolisant, the active ingredient in Wakix, as a treatment for idiopathic hypersomnia during the second half of this year. Idiopathic hypersomnia is a rare chronic sleep disorder characterized by excessive daytime sleepiness and difficulty waking up from sleep. About 10 of every 100,000 people are afflicted with the condition, according to an estimate by the National Institutes of Health

In addition, the company is working on a next-generation formulation of pitolisant, which the FDA has classified as a first-in-class histamine 3 receptor antagonist. If approved, Harmony said, the company's intellectual property protections for pitolisant could potentially extend to 2044.

Sources: Philadelphia Business Journal

Link to article: https://www.bizjournals.com/philadelphia/news/2024/06/24/harmony-biosciences-wakix-pediatric-narcolepsy.html?ana=brss_3970