Takeda (TAK.US) has received approval from the U.S. FDA for Oxybate for the treatment of type 1 narcolepsy.

On August 5 local time, the FDA announced that Takeda's Oveporexton has been approved for marketing in the United States for the treatment of type 1 narcolepsy (NT1). Previously, the product had received Breakthrough Therapy Designation and Priority Review from the FDA. In July of this year, Oveporexton was also approved for marketing in China. The press release indicated that this is the first drug approved for the treatment of type 1 narcolepsy, capable of alleviating all symptoms of the disease. It is also the first drug to act by directly targeting the orexin signaling pathway, which is disrupted in this condition. Narcolepsy is a rare chronic neurological disorder characterized by difficulties in maintaining wakefulness, primarily presenting as irresistible daytime excessive sleepiness (EDS), cataplexy, and other rapid eye movement (REM) sleep phenomena such as sleep paralysis and hypnagogic hallucinations. Type 1 narcolepsy, also known as cataplectic narcolepsy, results from the significant loss of orexin-producing neurons in the lateral hypothalamus, leading to a marked reduction in orexin levels and clinical symptoms characterized by excessive daytime sleepiness accompanied by cataplexy. Oveporexton (TAK-861, formerly known as Orebrelor) is an oral selective agonist of the orexin receptor 2 (OX2R). It selectively stimulates OX2R to restore the downstream neurotransmitter activity lost due to decreased endogenous orexin levels, addressing the underlying issue of orexin deficiency that contributes to type 1 narcolepsy.