FDA Approves First Drug to Treat a Cause of Narcolepsy

The U.S. Food and Drug Administration recently approved Orzeyful, the first drug to treat a cause of narcolepsy. Narcolepsy affects about 200,000 people in the U.S. and 3 million worldwide, with symptoms ranging from daytime sleepiness to sudden sleep episodes and loss of muscle control. There is currently no cure for narcolepsy, and treatment generally involves stimulant drugs, amphetamines, and behavior changes like scheduled naps.

Orzeyful targets the orexin pathway, which regulates the brain's sleep and wake cycles. Without the orexin, the brain loses its ability to control when the body sleeps and is awake. Orzeyful is particularly effective for narcolepsy type 1, which can be more dangerous due to muscle control loss and potential injury. The drug works by activating the dysfunctional orexin pathway, restoring the brain's alertness during the day.

Based on Takeda's studies involving over 270 patients, those taking Orzeyful were better able to stay awake during the day, had fewer cataplexy episodes, and experienced fewer other symptoms like irregular nighttime sleep and hallucinations. The FDA is now reviewing the drug for potential addictive properties, and Takeda CEO Julie Kim expects it to receive a schedule IV designation, indicating a low risk of dependence or abuse.

Developing Orzeyful took 15 years, with initial versions not practical due to the need for continuous IV medication. An earlier pill form showed promise but had liver abnormalities, leading to its discontinuation. However, Orzeyful proved safer, with no negative side effects reported in studies. This success could lead to treatments for other sleep disorders, with Takeda already researching another orexin-based drug for narcolepsy type 2 and idiopathic hypersomnia.