Harmony Biosciences today announced that the U.S. Food and Drug Administration denied the Citizen Petition filed by a short seller claiming that WAKIX is not safe and effective for the treatment of excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy.
FDA confirms the favorable benefit-risk profile of WAKIX Harmony Biosciences focused on continuing to advance its pipeline assets across three rare CNS franchises; anticipates at least one new product or indication launch annually over the next five years PLYMOUTH MEETING, Pa., June 25, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced that the U.S. Food and Drug Administration denied the Citizen Petition filed by a short seller claiming that WAKIX is not safe and effective for the treatment of excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The Agency’s decision to reject the Petition was anticipated and confirms the Company’s long-held position that the allegations in the Petition were unfounded and without merit. In its denial, the FDA rejected all three requests from the Petitioner including withdrawal for all indications, immediate alert distribution (‘Dear HCP Letter’) to prescribers, and transitioning to a Compassionate Use Program with a REMS protocol, and stated “the FDA determined that WAKIX has a favorable benefit-risk profile under its approved conditions of use, and your Petition did not provide information that changes that assessment.”1 The FDA denied the Petition after approving the supplemental New Drug Application (sNDA) for WAKIX on June 21, 2024, which expanded the indication for WAKIX to include the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy. The denial letter states, “the FDA has carefully considered the information submitted in the Petition, other data available to the Agency, and relevant published literature. Based on our review of these materials and for the reasons stated, the Petition is denied.”2 Harmony Biosciences stated: “We are pleased with the FDA’s decision to deny the short seller Petition filed in March 2023, which tried to cast doubt on a safe and effective treatment while deliberately attempting to impact our stock price for profit. We appreciate the Agency’s careful review of the unfounded claims in the Petition and its definitive action to deny the claims and resolve any potential doubt about the favorable benefit-risk profile of WAKIX. Looking ahead, we confirm our outlook that WAKIX represents a billion-dollar plus market opportunity in adult narcolepsy alone, and we are well on our way to achieving further growth in the pitolisant franchise. We are also on track toward filing an sNDA for WAKIX in idiopathic hypersomnia later this year (2H 2024). In anticipation of the confirmation of the favorable benefit-risk profile of WAKIX, we have already been working on the next-generation formulations of pitolisant to address unmet medical needs. These formulations are designed to provide meaningful improvement, such as an enhanced pharmacokinetic (PK) profile and higher dosage range to drive greater efficacy. These innovations are expected to generate new IP to extend the pitolisant franchise out beyond 2040. Beyond WAKIX, we have expanded our pipeline and diversified our portfolio resulting in three promising orphan rare CNS franchises in advanced stages of development, each with the potential to generate $1 billion to $2 billion in peak sales. These franchises are protected by patents extending into the late 2030s to mid-2040s. As we continue to execute our growth strategy, we are now poised to lead as a patient-centered CNS biotechnology company, providing innovative treatments for patients with unmet medical needs while driving substantial and durable value creation. Our pipeline consists of eight assets being studied across 13 development programs within these three CNS franchises. Three of our development programs are in Phase 3 trials and we have two Phase 3 ready assets that we are working on. With this late-stage pipeline, we anticipate launching at least one new product or indication annually over the next five years and have the potential to bring new treatment options to hundreds of thousands of people living with rare diseases.” The FDA’s detailed response letter can be found at the following link: https://downloads.regulations.gov/FDA-2023-P-1273-0005/attachment_1.pdf. 1 FDA Response Letter, Docket No: FDA-2023-P-1273, Page 12, Paragraph 3, Sentence 3. About Narcolepsy About WAKIX® (pitolisant) Tablets INDICATIONS AND USAGE IMPORTANT SAFETY INFORMATION Contraindications Warnings and Precautions The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is contraindicated in patients with severe hepatic impairment and not recommended in patients with end-stage renal disease (ESRD). Adverse Reactions 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) for WAKIX were headache (19%) and insomnia (7%). The overall adverse reaction profile of WAKIX in the pediatric clinical trial was similar to that seen in the adult clinical trial program. Drug Interactions Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required. H1 receptor antagonists that cross the blood-brain barrier may reduce the effectiveness of WAKIX. Patients should avoid centrally acting H1 receptor antagonists. WAKIX is a borderline/weak inducer of CYP3A4. WAKIX may reduce the effectiveness of sensitive CYP3A4 substrates, including hormonal contraceptives. Patients using hormonal contraception should be advised to use an alternative non-hormonal contraceptive method during treatment with WAKIX and for at least 21 days after discontinuing treatment. Use in Specific Populations The safety and effectiveness of WAKIX have not been established for treatment of excessive daytime sleepiness in pediatric patients less than 6 years of age with narcolepsy. The safety and effectiveness of WAKIX have not been established for treatment of cataplexy in pediatric patients with narcolepsy. WAKIX is extensively metabolized by the liver. WAKIX is contraindicated in patients with severe hepatic impairment. Dosage adjustment is recommended in patients with moderate hepatic impairment. WAKIX is not recommended in patients with end-stage renal disease. Dosage adjustment of WAKIX is recommended in patients with eGFR <60 mL/minute/1.73 m2. Dosage reduction is recommended in patients known to be poor CYP2D6 metabolizers; these patients have higher concentrations of WAKIX than normal CYP2D6 metabolizers. Please see the Full Prescribing Information for WAKIX for more information. To report suspected adverse reactions, contact Harmony Biosciences at 1-800-833-7460 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. About Harmony Biosciences Forward Looking Statement Harmony Biosciences Investor Contact: Harmony Biosciences Media Contact: View original content to download multimedia:https://www.prnewswire.com/news-releases/harmony-biosciences-acknowledges-us-food--drug-administration-fda-action-denying-the-citizen-petition-for-wakix-pitolsiant-302181609.html SOURCE Harmony Biosciences | ||
Company Codes: NASDAQ-NMS:HRMY |