Takeda (TAK.US) Orexin Approved by US FDA for the Treatment of Type 1 Narcolepsy

According to Golden Ten Data APP, on August 5 local time, the FDA announced that Takeda (TAK.US)'s Oveporexton tablets have been approved by the US FDA for the treatment of type 1 narcolepsy (NT1). Previously, this product had been granted Breakthrough Therapy Designation and Priority Review status by the FDA. In July of this year, Oveporexton was approved for market launch in China.

The press release shows that this is the first drug approved for the treatment of type 1 narcolepsy, capable of alleviating all symptoms of the disease, and also the first drug to work by directly targeting the loss of the orexin signaling pathway that causes this condition.

Narcolepsy is a rare chronic neurological disorder characterized by difficulty maintaining sleep-wake cycles. Its main clinical features are uncontrollable excessive daytime sleepiness (EDS), cataplexy, and other rapid eye movement (REM) phenomena such as sleep paralysis and hypnagogic hallucinations. Type 1 narcolepsy, also known as cataplexy-type narcolepsy, is caused by a significant loss of orexin-producing neurons in the lateral hypothalamus, resulting in a marked reduction of orexin. Clinical symptoms include excessive daytime sleepiness accompanied by cataplexy episodes.

Oveporexton (TAK-861, formerly known as Oboreton) is an oral orexin receptor 2 (OX2R) selective agonist. It selectively stimulates OX2R to restore the activity of downstream neurotransmitters lost due to decreased endogenous orexin levels, addressing the underlying orexin deficiency that leads to type 1 narcolepsy.