Takeda has secured FDA approval for Orzeyful, a twice-daily tablet treating narcolepsy type 1 in adults, making it the first medicine of its kind.
Unlike existing narcolepsy treatments that address symptoms, Orzeyful targets the root cause of the disorder by mimicking the body’s natural orexin cycle.
Orexin is a neuropeptide that helps regulate wakefulness, appetite, and energy, and is critically deficient in patients with narcolepsy type 1.
The approval covers oveporexton, which becomes the first orexin receptor agonist to reach the market as a treatment for this debilitating neurological condition.
Narcolepsy type 1 is a rare, chronic disorder estimated to affect around 120,000 people in the United States, causing excessive daytime sleepiness, cataplexy, and disrupted sleep.
“The FDA approval of Orzeyful marks a new chapter for the narcolepsy type 1 community, as we introduce an entirely new class of medicine that will potentially redefine how this disease is managed and how people feel on treatment,” Takeda CEO Julie Kim said in a statement.
Takeda has significant commercial expectations for Orzeyful, with the company pegging peak sales potential at between $2 billion and $3 billion.
The approval follows Phase 3 clinical trials, including the global FirstLight and RadiantLight studies, which showed Orzeyful significantly reduced cataplexy attacks and improved daytime sleepiness compared to placebo.
“Orzeyful is just the beginning. With orexin emerging as a powerful therapeutic pathway, Takeda is unlocking the potential of this new class of medicine for patients across a range of disorders,” said Andy Plump, M.D., Ph.D., Takeda’s R&D chief.
The drug will become available to patients later this year, following a scheduling review process with the U.S. Drug Enforcement Administration.
Takeda is also awaiting an FDA decision on rusfertide, a hepcidin-mimetic peptide being reviewed for the blood disorder polycythemia vera, with peak sales projected between $1 billion and $2 billion.
Rusfertide was acquired through a $300 million licensing deal with Protagonist Therapeutics in 2024 and posted positive results from a Phase 3 trial last year.
Beyond these two candidates, Takeda’s pipeline ambitions are also centred on zasocitinib, a follow-on to its established therapy Entyvio.