[Shanghai, Dec 22, 2023] – Today, RareStone Group announced the inclusion of Wakix® (Pitolisant) in China’s National Reimbursed Drug List (NRDL), marking a significant milestone in enhancing access to innovative medicines for narcolepsy in China.
Narcolepsy, a globally recognized rare disease, was added to China’s "Second Batch of Rare Disease Catalog" in September 2023. It presents as a rare sleep/wake disorder typically experienced throughout life, often starting in childhood (around 8 to 10 years old). Clinically, it manifests primarily through core symptoms such as excessive daytime sleepiness (EDS), cataplexy, hypnagogic hallucinations, sleep paralysis, and disrupted nighttime sleep.
This condition significantly impacts the learning/work capabilities and overall well-being of affected individuals, elevating the risk of accidents due to excessive sleepiness and sudden collapses. According to a 2022 local narcolepsy research study in China, the risk of hospitalization due to accidents, such as traffic incidents, is 6.7 times higher among patients compared to healthy individuals. Additionally, 43% of adult patients have completed only high school or lower education, resulting in a 30% decrease in employment rates compared to non-afflicted individuals.
Wakix® (Pitolisant) stands as the world's first approved selective histamine H3 receptor inverse agonist drug. It has been granted orphan drug designation by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA), receiving breakthrough therapy designation from the FDA. In China, on June 30, 2023, the National Medical Products Administration (NMPA) officially approved Pitolisant (Wakix®) for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy.
Pitolisant represents the first and only approved therapeutic option for narcolepsy in China. It is a non-controlled substance globally and stands as the only first-line treatment option, as recommended by all major clinical guidelines in the United States, Europe, and China, for single-drug usage to manage multiple core symptoms of narcolepsy.
Extensive studies have revealed Pitolisant's significant efficacy and reliable safety. Globally conducted clinical research demonstrates Pitolisant's notable effects in improving control of excessive daytime sleepiness (-33% vs. -17%) and reducing weekly cataplexy attacks (-64% vs. -7%). The likelihood of improving ESS scores is nine times higher in the treatment group compared to the placebo. Real-world studies in China from 2021 to 2022 have affirmed Pitolisant's effectiveness in improving excessive daytime sleepiness (-33%) and reducing daily cataplexy episodes (-51%), aligning well with global research findings.
Pitolisant stands as the only non-controlled substance for narcolepsy treatment worldwide, posing no risk of withdrawal syndrome upon drug discontinuation. With the majority of adverse drug reactions occurring in less than 2% of cases, it has accumulated safety data from over 3,000 clinical trial participants over seven years since its global launch, more than 36,000 patient-years post-launch, and up to five years of follow-up data, affirming its excellent tolerability and long-term safety. Unlike other narcolepsy treatment options, it has never received any safety warnings or black box warnings from regulatory authorities worldwide. The real-world study of Pitolisant use in Bo’ao, China, found no severe adverse events reported, with recorded adverse events being mild to moderate, consistent with known safety data overseas. Not requiring special management as a controlled substance, Pitolisant lessens patient follow-up burden and reduces healthcare system management costs too.
Pitolisant has consistently received recommendations from authoritative guidelines both domestically and internationally for single-drug use as a first-line treatment option to control multiple core symptoms of narcolepsy. The "Chinese Narcolepsy Diagnosis and Treatment Guidelines (2022 Edition)" strongly endorse Pitolisant as the first-choice medication, receiving the highest recommendations and evidence levels for multiple core symptoms. Additionally, U.S. guidelines strongly recommend Pitolisant, and European guidelines and expert consensus explicitly support its use as a first-line single-drug treatment choice.
Statements from Chinese Patient and Physician Experts
Ms. Bao, Mindong
Founder of "DreamHouse," the largest narcolepsy patient organization in China
“In the past six months, there's been a series of good news for narcolepsy patients in China! In June 2023, Pitolisant, the first drug (for narcolepsy) in China, was approved by the National Medical Products Administration. In September, narcolepsy was included in the 'Second Batch of Rare Disease Catalog' of China. Now in December, Pitolisant is reimbursed and included in the new version of the National Reimbursed Drug List, achieving a great breakthrough for narcolepsy drug access in China! Many thanks to the attention and support from various government agencies and the industry to rare diseases like narcolepsy! Looking ahead, we hope that insurance coverage can be swiftly implemented, allowing more patients to access innovative drug treatments and receive actual reimbursement support."
Professor Han, Fang
Chairman of the Asian Sleep Association, Secretary-General of the World Sleep Society
Sleep Medicine Department, Peking University People's Hospital
“Narcolepsy is a rare sleep disorder highly prevalent during school age. Clinical symptoms like excessive daytime sleepiness and sudden collapses severely impact patients' learning, life, and social functions, leading to difficulties in receiving proper education, employment challenges, and reduced income. Pitolisant, as the first approved medication for narcolepsy in the country and now included in the National Reimbursed Drug List, will provide much wider access to this new drug and allow much more patients to receive early diagnosis and proper treatment, and even helping narcolepsy patients to reintegrate into society.”
Professor Zhao, Zhongxin
Honorary Chairman of the Sleep Rehabilitation Special Committee, Chinese Society of Rehabilitation Medicine
Neurology Department, Shanghai Changzheng Hospital, Naval Medical University
“Narcolepsy currently cannot be cured and require long-term treatment to control the serious impact on learning, work, and life of patients. Pitolisant is recommended as a first-line therapy by narcolepsy guidelines in China, the U.S., and Europe, and is the only non-controlled substance medication. Its newly granted reimbursement status in China well demonstrates the increasingly greater attention and significance the National Reimbursement Authority places on rare disease medications, offering a boon to narcolepsy patients by significantly reducing cost burdens. It also helps addressing the current issues of 'off-label' drug use and difficulties in obtaining controlled substance prescriptions which leads to poor treatment compliance.
Professor Zhan, Shuqin
Vice Chairman of the Chinese Sleep Research Society,
Director of the Sleep Disorders Committee,
Chief Physician, Department of Neurology, Xuanwu Hospital, Capital Medical University
“Since May 2021, Pitolisant has been used among Chinese patients at hospitals in Boao, Hainan Province. On June 30, 2023, it received approval from the National Medical Products Administration. On September 20, 2023, narcolepsy was included in the Second Batch of Rare Disease Catalog. Finally, on December 13, 2023, it won reimbursement status in China. These milestones illustrate the increasing focus the whole country is placing on rare diseases and narcolepsy. Each milestone event will propel diagnosis and treatment of narcolepsy in China into a new stage, bringing more hope to patients and their families.
RareStone’s Voice Dr. Xiang, Yu CEO, RareStone Group
"Wakix® achieving reimbursement status in China in 2023, just a few months after receiving approval in this country, showcases the China as a country is paying more special attention to the rare disease groups. It’s encouraging to see China’s new value-oriented, evidence-based approach to health insurance payments is put to work. RareStone will continue its commitment to Chinese rare disease community, and put even more efforts in the diagnosis, treatment, and ecosystem building for narcolepsy in China, helping more patients to benefit from innovative treatments.”