Tokyo, Japan, December 26, 2024 — Aculys Pharma, Inc. (“Aculys”), a clinical stage biopharmaceutical company focused on commercializing innovative treatments for neurological and psychiatry conditions, has announced that pitolisant (“this drug”), a histamine H3 receptor antagonist/inverse agonist for narcolepsy, which is currently under development, has received orphan drug designation from the Ministry of Health, Labour and Welfare. This drug achieved its primary efficacy endpoint in the domestic Phase 3 clinical trial, demonstrating results consistent with global studies to date. Based on this data, Aculys plans to submit a New Drug Application for pitolisant to help create a world where narcolepsy patients and their families can live with greater peace of mind and authenticity. Orphan drug designation expedites the regulatory review of a New Drug Application from 12 months to nine (9) months and also provides additional benefits (for more details, please refer to the “About Orphan Drugs”).
Hidemasa Tanigaki, Chief Executive Officer of Aculys, stated, “Patients with narcolepsy face serious challenges in their daily lives due to symptoms that occur against their own volition. We are delighted to have received orphan drug designation for this drug when there has been no new treatment option for narcolepsy in Japan for almost 2 decades. We are committed to delivering this drug to narcolepsy patients as soon as possible.”
View details of the clinical trial at: https://jrct.niph.go.jp/en-latest-detail/jRCT2031220432
■ About Orphan Drugs
Based on Article 77-2 of the Pharmaceutical and Medical Device Act, the Minister of Health, Labour and Welfare designates drugs which meet the conditions that the number of eligible patients in Japan is less than 50,000 and that there is a particularly high medical need. In doing so, it refers to the opinions of the Pharmaceutical Affairs and Food Sanitation Council. If a drug is so designated, companies will be able to receive support measures such as grants, tax measures, priority examinations, and extensions of reexamination periods. For details of this designation system, please see the Ministry of Health, Labour and Welfare’s website. (https://www.mhlw.go.jp/stf/seisakunitsuite/bunya/0000068484.html)
■ About pitolisant
Pitolisant is an antagonist/inverse agonist that selectively binds to the histamine H3 receptor, an autoreceptor located in the presynaptic region of the histamine-containing neurons in the human brain that plays a critical role in regulating sleep and wake rhythm. The drug was discovered by Paris-based Bioprojet and in Europe it was approved by the European Medicines Agency (EMA) for the treatment of narcolepsy with or without cataplexy in 2016 and for the treatment of excessive daytime sleepiness associated with obstructive sleep apnea syndrome (OSAS) in 2021. In the United States, the drug was approved by the Food and Drug Administration (FDA) for the treatment of excessive daytime sleepiness associated with narcolepsy in 2019 and cataplexy associated with narcolepsy in 2020 and is already used in the clinical setting. As of the end of 2023, pitolisant has obtained regulatory approval in 38 countries including the US and EU for narcolepsy, and in 30 countries in Europe for OSAS.
■ About narcolepsy
Patients with narcolepsy often start suffering from symptoms in childhood, adolescence, or young adulthood, but are not diagnosed until much later due to misdiagnosis and low awareness of the condition’s symptoms, posing a global challenge.1 The causes and pathophysiology of narcolepsy are largely unknown. In healthy people, neurotransmitters in the brain, such as orexin and histamine, help maintain the regular cycle of daytime wakefulness and nighttime sleep (REM and non-REM sleep), while in patients with narcolepsy, this sleep-wake cycle becomes irregular.2 Due to this irregular sleep-wake cycle, patients with narcolepsy suddenly, and against their will, fall asleep or have difficulty maintaining concentration while engaged in school or work. Some patients also have cataplexy attacks, which involve sudden loss of muscle tone of the whole body or certain voluntary muscles that are triggered by strong emotions, hypnagogic hallucinations (a hallucination that occurs right before falling asleep), and sleep paralysis (“old hag” syndrome). Different patients experience different types and severity of symptoms, but these symptoms significantly affect patients’ lifestyles, including challenges in education or employment.3
■ About Aculys Pharma, Inc.
Aculys Pharma is a clinical stage biopharmaceutical company that is pioneering ways to eliminate drug lag/drug loss in Japan, and is working to resolve social issues related to neurological and psychiatry diseases. Its corporate name was created from the philosophy of serving as a “Catalyst to Access.” Aiming to act as a bridge for innovative medical care in the field of neuropsychiatry, Aculys Pharma develops and commercializes novel pharmaceuticals introduced from the US and European countries and provides innovations for better medical care to patients, their families, healthcare professionals, and society. Aculys Pharma is based in Tokyo, Japan, and was founded by Catalys Pacific.
Company name: Aculys Pharma, Inc.
Address: 2-14-4 Kita-Aoyama, Minato-ku, Tokyo, Japan
Representative: Hidemasa Tanigaki
Establishment date: January 2021
URL: https://aculys.com
Information concerning pharmaceutical products (compounds under development) contained herein is not intended as advertising or as medical advice but for disclosure of management information.
References:
- Thorpy MJ, Krieger AC. Delayed diagnosis of narcolepsy: characterization and impact. 2014 May;15(5):502-7.
- Scammell TE. Narcolepsy. NEJM. 2015;373:2654-2662.
- Scammell TE, Jackson AC, Franks NP, Wisden W, Dauvilliers Y. Histamine: neural circuits and new medications. Sleep. 2019 Jan 1;42(1):zsy183.