Home / Health / FDA Approves First Narcolepsy Drug Targeting Orexin
FDA Approves First Narcolepsy Drug Targeting Orexin
6 Aug
Summary
- FDA approved Takeda's new oral drug for narcolepsy type 1.
- The drug, Orzeyful, targets the loss of orexin signaling.
- It is the first treatment to address all symptoms of the disorder.
The U.S. Food and Drug Administration has given its approval to Takeda Pharmaceuticals' new oral medication, Orzeyful, for the treatment of narcolepsy type 1. This groundbreaking approval signifies the availability of the first medication designed to address all facets of the rare sleep disorder.
Narcolepsy type 1 is characterized by the loss of specific brain cells responsible for producing orexin, a crucial chemical messenger that regulates wakefulness and sleep cycles. This condition, affecting approximately 120,000 individuals in the United States, leads to excessive daytime sleepiness, sudden muscle tone loss, cognitive issues, and disrupted nighttime sleep.
Orzeyful, administered as a twice-daily pill, directly targets the orexin signaling deficit that underlies the disease. Clinical studies demonstrated that patients receiving Orzeyful showed significant improvements in their ability to maintain wakefulness during the day compared to those on a placebo. This approval follows data from two key studies.
Previously, narcolepsy type 1 lacked a unified treatment approach, and no existing medications specifically targeted the orexin system. The drug's classification as a controlled substance is currently under review by the Drug Enforcement Administration, with a ruling anticipated within 90 days. Following this determination, Takeda plans to make Orzeyful available through a specialty pharmacy.
