FDA Grants Breakthrough Therapy Designation to Takeda’s Experimental Narcolepsy Drug

The U.S. Food and Drug Administration granted Takeda Pharmaceutical Breakthrough Therapy designation to TAK-994,1 its phase 2 experimental oral orexin agonist treatment for narcolepsy type 1, a rare neurologic disorder characterized by excessive daytime sleepiness.

Photo: Sarah Sheikh, head of the Neuroscience Therapeutic Area Unit at Takeda

Narcolepsy type 1 (NT1), is a chronic neurological disorder that alters the sleep-wake cycle. Excessive daytime sleepiness is a hallmark symptom of NT1 and is characterized by a person’s inability to stay awake and alert throughout the day and falling asleep unintentionally or at inappropriate times on a daily basis.

The FDA’s Breakthrough Therapy designation process is designed to expedite the development and review of a drug that is intended to treat a serious condition, for which preliminary clinical evidence exists indicating it may demonstrate a substantial improvement over available therapies on at least one clinically significant endpoint.

TAK-994 is designed to selectively target orexin 2 receptors. It is currently being studied for the treatment of excessive daytime sleepiness (EDS) in patients with NT1.

The Breakthrough Therapy designation was based, in part, on early phase and preliminary clinical data that indicates TAK-994 may demonstrate substantially improved objective and subjective measurements of daytime wakefulness in NT1 patients. Currently, TAK-994 is being studied in an ongoing phase 2 study.

“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,” said Sarah Sheikh, head of the Neuroscience Therapeutic Area Unit at Takeda. “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.”

Author: Rare Daily Staff