PLYMOUTH MEETING, Pa. - Harmony Biosciences Holdings , Inc. (NASDAQ:HRMY) has received confirmation from the U.S. Food and Drug Administration (FDA) that its narcolepsy medication, WAKIX, maintains a favorable benefit-risk profile. The FDA decision comes in response to a Citizen Petition filed by a short seller, which the Agency has now denied.
The petition, submitted in March 2023, suggested that WAKIX was not safe or effective for treating excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The FDA's denial of the petition follows the approval of a supplemental New Drug Application (sNDA) for WAKIX on June 21, 2024, allowing its use in pediatric patients aged six and older with narcolepsy.
In their response, the FDA stated that they found no new information in the petition to alter their assessment of the drug's safety and efficacy. The Agency also declined the petitioner's requests for WAKIX's withdrawal, immediate alert distribution to prescribers, and transition to a Compassionate Use Program with a Risk Evaluation and Mitigation Strategy (REMS) protocol.
Harmony Biosciences has expressed satisfaction with the FDA's decision, emphasizing the unfounded nature of the allegations and the drug's continued promise as a treatment for narcolepsy. The company anticipates further growth in the pitolisant franchise, including the filing of an sNDA for idiopathic hypersomnia later in the second half of 2024. Harmony is also working on next-generation formulations of pitolisant to extend the product's patent life beyond 2040.
Looking beyond WAKIX, Harmony Biosciences has expanded its pipeline to include three rare CNS franchises, each potentially capable of generating between $1 billion to $2 billion in peak sales. With a focus on innovative treatments for patients with unmet medical needs, the company plans to launch at least one new product or indication annually over the next five years.
Narcolepsy affects approximately 170,000 Americans and is characterized by excessive daytime sleepiness, among other symptoms. WAKIX, a first-in-class medication, operates as a selective histamine 3 (H₃) receptor antagonist/inverse agonist, although its exact mechanism of action is not fully understood.
The information in this article is based on a press release statement from Harmony Biosciences.
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