Harmony Biosciences Acknowledges FDA Denial of Wakix® (Pitolisant) Petition

Harmony Biosciences Holdings, Inc. has announced that the U.S. Food and Drug Administration (FDA) has denied a Citizen Petition filed by a short seller who claimed that WAKIX is neither safe nor effective for treating excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The FDA's decision to reject the petition aligns with Harmony Biosciences’ long-standing assertion that the allegations were baseless.

The FDA's denial addressed all three requests from the petitioner: the withdrawal of WAKIX for all indications, the immediate distribution of a ‘Dear HCP Letter’ to prescribers, and the transition of WAKIX to a Compassionate Use Program with a REMS protocol. The FDA stated, “WAKIX has a favorable benefit-risk profile for its approved conditions of use, and the petition did not provide information that changes that assessment.”

The FDA's decision came shortly after it approved a supplemental New Drug Application (sNDA) for WAKIX on June 21, 2024. This approval expanded WAKIX's indication to include the treatment of EDS in pediatric patients aged six and older with narcolepsy. In their denial letter, the FDA emphasized that it had carefully reviewed the information in the petition, along with other data available to the agency, and found no reason to alter its positive assessment of WAKIX.

Harmony Biosciences expressed satisfaction with the FDA’s decision, underscoring the unfounded nature of the petition which aimed to manipulate the company’s stock price for profit. The company reaffirmed its belief in the significant market potential of WAKIX, particularly in adult narcolepsy, projecting a billion-dollar market opportunity. Harmony is also preparing to file another sNDA for WAKIX in idiopathic hypersomnia later this year. The company has been working on next-generation formulations of pitolisant, WAKIX's active ingredient, to address unmet medical needs and improve efficacy, potentially extending the drug's intellectual property protection through the 2040s.

Apart from WAKIX, Harmony Biosciences has diversified its pipeline, focusing on three promising rare central nervous system (CNS) franchises that are in advanced stages of development. Each franchise has the potential to achieve peak sales between $1 billion and $2 billion, supported by patents extending into the late 2030s to mid-2040s. The company's growth strategy involves the potential launch of at least one new product or indication annually over the next five years, with multiple assets in Phase 3 trials and two more ready for Phase 3.

WAKIX, approved for treating EDS and cataplexy in adults with narcolepsy and EDS in pediatric patients aged six and older, is a groundbreaking medication developed in France by Bioprojet. It works by acting as a selective histamine 3 (H₃) receptor antagonist/inverse agonist, although its exact mechanism is unclear. WAKIX has been granted orphan drug designation and breakthrough therapy designation for its potential in treating narcolepsy and cataplexy.

The drug carries specific safety information, including contraindications for patients with severe hepatic impairment and a history of hypersensitivity to pitolisant. WAKIX can prolong the QT interval, necessitating caution in patients with known QT prolongation or cardiac arrhythmias. The most common adverse reactions include insomnia, nausea, and anxiety in adults, and headache and insomnia in pediatric patients.

Harmony Biosciences specializes in developing treatments for rare neurological diseases. Established in 2017, the company focuses on translating innovative science into therapeutic options for patients with unmet medical needs, driven by a commitment to improving lives through empathetic and innovative approaches.