Takeda's Investigational Oral Orexin Agonist TAK-994 Receives FDA Breakthrough Therapy Designation for Narcolepsy Type 1

Takeda recently announced that the U.S. FDA has granted Breakthrough Therapy Designation to its investigational oral orexin agonist TAK-994 for the treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy type 1 (NT1). NT1 is a chronic neurological disorder that disrupts the sleep-wake cycle. EDS is a hallmark symptom of NT1, characterized by patients' inability to maintain wakefulness and alertness throughout the day, resulting in sleep onset at inappropriate times. TAK-994 is designed to selectively target the orexin 2 receptor and is currently being evaluated in a Phase 2 clinical trial.

Studies have shown that patients with narcolepsy type 1 (NT1) exhibit a significant loss of orexin-producing neurons in the brain; therefore, orexin 2 receptor agonists may replace endogenous orexin and activate wake-promoting signaling pathways.

This breakthrough therapy designation was granted based on early and preliminary clinical data indicating that Takeda’s investigational oral orexin agonist significantly improves objective and subjective measures of daytime wakefulness in patients with narcolepsy type 1 (NT1). Data from the Phase 2 clinical trial will be presented at upcoming scientific conferences upon completion of the study.

Previously, the intravenous formulation of this drug, TAK-925, demonstrated efficacy in maintaining wakefulness in patients with NT1 in a Phase 1 proof-of-concept clinical trial.

▲TAK-925 demonstrated promising activity in a proof-of-concept trial (Image source: Takeda official website)

Note: The original text has been abridged.

References:

[1] 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). Retrieved August 1, 2021, fromhttps://www.businesswire.com/news/home/20210728005256/en

[2] Scanmmell (2001). Wakefulness: An eye-opening perspective on orexin neurons. Current Biology, DOI:https://doi.org/10.1016/S0960-9822(01)00466-3

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