Japan approves new therapy for narcolepsy, sleep apnea-related EDS
Newly cleared treatment is not classified as a controlled substance
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Japan has approved Wakix (pitolisant) for narcolepsy and excessive daytime sleepiness associated with obstructive sleep apnea syndrome. (Image from iStock)
Japan’s Ministry of Health, Labour and Welfare has approved Wakix (pitolisant), a once-daily oral treatment, for narcolepsy types 1 and 2. Wakix is used to treat excessive daytime sleepiness (EDS) and cataplexy in people with narcolepsy.
The country also approved the first-in-class therapy for EDS associated with obstructive sleep apnea syndrome (OSAS) in patients receiving treatment for airway obstruction.
Wakix works through the brain’s histamine system and is not classified as a controlled substance.
Wakix targets histamine system to promote wakefulness
“This milestone marks an important advancement for sleep medicine in Japan and brings forward a treatment option that is not classified as a controlled substance,” Philippe Martin, chief R&D officer at Viatris, which holds the license to market the therapy in Japan, said in a company press release.
Narcolepsy is a chronic neurological disorder marked by EDS and other sleep-related, cognitive, and mood symptoms. There are two types. Type 1, generally the more severe form, involves cataplexy — a sudden, brief loss of muscle tone. Type 2 may be less severe and usually does not involve cataplexy.
OSAS, a separate condition involving the obstruction of airways during sleep, can also be associated with EDS, which may persist even in patients already being treated for the underlying airway problem, including those on continuous positive airway pressure (CPAP) therapy.
EDS is a symptom shared by both conditions and represents an unmet medical need in Japan. It can interfere with daily functioning, work productivity, and quality of life.
Narcolepsy treatments can include traditional stimulants that increase alertness and ease EDS. Wakix works differently and is not classified as a controlled substance.
Instead, Wakix is a histamine H3 receptor antagonist/inverse agonist, which blocks or reduces H3 receptor activity in the brain, increasing levels of histamine, a signaling molecule that promotes wakefulness. This is expected to help stabilize sleep-wake transitions, easing EDS and reducing cataplexy.
Wakix was discovered by France-based Bioprojet and is licensed to Viatris for the Japanese market. The drug has already received approval for narcolepsy in more than 38 countries, including the U.S. and multiple countries in Europe, and for OSAS-related EDS in more than 29 European countries.
Japan approval supported by Phase 3 data
In Japan, Wakix has been designated an orphan drug for narcolepsy, a status that helps support the development of therapies for rare diseases.
The therapy’s approval was supported by Phase 3 clinical trials conducted in Japan and multiple international clinical studies that evaluated Wakix in patients with narcolepsy and in those with OSAS who continued to experience EDS despite CPAP treatment.
Although the announcement didn’t provide details of the clinical trial results in Japan, the Harmony1 study (NCT01067222), conducted in Europe, showed that Wakix treatment eased patient-reported sleepiness among adults with narcolepsy compared with a placebo. Among participants with cataplexy, it also reduced the daily rate of cataplexy attacks. Similar benefits were reported in other Phase 3 studies.
“As Japan’s first approved histamine H3 receptor antagonist/inverse agonist for these conditions, WAKIX offers a much-needed novel treatment option for patients,” Martin said.
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