FDA Grants Breakthrough Therapy Designation for Narcolepsy Drug

The FDA has granted Breakthrough Therapy Designation (BTD) to Takeda Pharmaceutical’s investigational orexin agonist, TAK-994, for narcolepsy.

The pharmaceutical marker is studying the medication for the treatment of patients with excessive daytime sleepiness (EDS) in patients with narcolepsy type 1 (NT1), a chronic neurological disorder that alters the sleep-wake cycle, company officials said in a news release.

The incidence of narcolepsy is about 1 in 2,000, and most researchers believe that the disorder remains undiagnosed or misdiagnosed in many affected individuals. There is increasing evidence that narcolepsy is an autoimmune disorder, according to the National Organization for Rare Disorders.

“Individuals with narcolepsy type 1 suffer from excessive daytime sleepiness, which might mean routinely falling asleep at work or in school. If approved, investigational TAK-994 has the potential to transform the way we currently treat NT1 by addressing the underlying orexin deficiency central to the disease,” Sarah Sheikh, head of the Neuroscience Therapeutic Area Unit at Takeda, said in a statement.

Orexin plays a critical role in regulating a person's sleep wake cycle and supports the body's natural wake promoting pathways in the brain, Sheikh added.

FDA’s Breakthrough Therapy Designation was based in part on early phase and preliminary clinical data that indicates Takeda’s investigational oral orexin agonist may demonstrate substantially improved objective and subjective measurements of daytime wakefulness in NT1 patients.

Data from TAK-994’s current phase 2 study will be presented at a future scientific conference after the study’s conclusion.

Investigational TAK-994 was discovered at Takeda’s laboratories in Shonan, Japan, part of the open innovation center at the Shonan Health Innovation Park.

The global narcolepsy drug market is expected to reach nearly $4.6 billion by the end of 2026, according to 360 Research Reports’ Narcolepsy Drug Market report.