FDA Grants Priority Review to Takeda Narcolepsy Drug

Takeda has taken a major step toward introducing a novel treatment for narcolepsy type 1 after the U.S. Food and Drug Administration accepted its New Drug Application and granted Priority Review for oveporexton (TAK-861), an investigational oral therapy designed to address the root cause of the disease.

The FDA assigned a Prescription Drug User Fee Act (PDUFA) target decision date in the third quarter of this year. If approved, oveporexton could become the first therapy that directly restores orexin signaling, potentially transforming treatment for people living with narcolepsy type 1 (NT1).

NT1 is a chronic neurological disorder caused by the loss of orexin — a neurotransmitter that regulates wakefulness. Patients experience excessive daytime sleepiness and cataplexy, a sudden loss of muscle tone triggered by emotions. The disease can severely affect daily functioning, education, employment and social interaction. While current medications manage symptoms, many patients continue to struggle with persistent impairment.

Oveporexton is an orexin receptor 2–selective agonist designed to compensate for the missing orexin pathway rather than simply managing symptoms. According to Takeda, the regulatory submission is supported by data from the global Phase 3 FirstLight and RadiantLight trials.

Results from the studies showed statistically significant and clinically meaningful improvements across multiple measures, including wakefulness, daytime sleepiness, cataplexy frequency, attention, and overall quality of life. Patients achieved near-normal ranges across a broad spectrum of symptoms. The therapy was generally well tolerated, with insomnia, urinary urgency and urinary frequency reported as the most common adverse events.

Takeda’s head of research and development, Andy Plump, said the review represents an important milestone for the narcolepsy community, noting that the treatment approach aims to correct the underlying biological deficiency rather than provide temporary symptom relief.

The drug has already received Breakthrough Therapy designation from the FDA and China’s drug regulator, as well as Japan’s Sakigake designation, highlighting global regulatory recognition of its potential. Takeda said the filing will not affect its financial outlook for the fiscal year ending March 2026.

If approved, oveporexton could mark a paradigm shift in narcolepsy care by offering a disease-modifying therapy for a condition that has long lacked treatments targeting its biological cause.