Oral Orexin Agonist Gets Breakthrough Therapy Status for Narcolepsy Type 1

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The Company is currently investigating TAK-994 in adults with narcolepsy type 1 or type 2 in a phase 2 study.

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to TAK-994 for the treatment of excessive daytime sleepiness in patients with narcolepsy type 1.

TAK-994 is an investigational oral orexin agonist designed to selectively target orexin 2 receptors. The Company is currently investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of TAK-994 in adults with narcolepsy type 1 or type 2 in a phase 2 study (ClinicalTrials.gov Identifier: NCT04096560). Data from the completed study will be presented at a future scientific conference.

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

The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to therapies that are expected to have an impact on factors such as survival and daily functioning.


US Food and Drug Administration grants Breakthrough Therapy designation to Takeda’s investigational compound, TAK-994, an oral orexin agonist in clinical development for narcolepsy type 1 (NT1). News release. Takeda Pharmaceutical Company Limited. Accessed July 28, 2021.gshttps://www.businesswire.com/news/home/20210728005256/en/U.S.-Food-and-Drug-Administration-Grants-Breakthrough-Therapy-Designation-to-Takeda%E2%80%99s-Investigational-Compound-TAK-994-an-Oral-Orexin-Agonist-in-Clinical-Development-for-Narcolepsy-Type-1-NT1