FDA approves first drug treating root cause of narcolepsy type-1
Washington: A Japanese pharmaceutical company has received US FDA approval for the first drug that treats the root cause of narcolepsy type-1.
Called Orzeyful and developed by Takeda, the drug mimics orexin, a compound that regulates wakefulness that is disrupted in patients with the condition. Narcolepsy type-1 affects about 120,000 people in the US and causes intense daytime drowsiness, disrupted nighttime sleep and cataplexy — sudden muscle weakness while awake.
Traditionally treated with stimulants and nervous system depressants, Orzeyful is the first medicine to target the underlying biology.”For too long, people with narcolepsy type 1 have had to manage a complex, lifelong condition with treatments that only address pieces of it,” said Dr. Tiffany Farchione, director of the FDA’s Division of Psychiatry, in an August 5 statement. “This new drug is the first medicine that impacts the underlying biology of the disease.”Final trials involving 273 patients showed those taking the twice-daily pill for 12 weeks stayed awake longer during the day and slept better at night compared to placebo.”It’s the best thing that has ever happened to me. My quality of life is so much better,” said trial participant Tyler Chapman.
Common side effects included increased urination frequency and insomnia, though incidence was considered low.
Takeda said the approval opens research into orexin as a therapeutic pathway for other neurological conditions including ADHD and Parkinson’s.”The FDA approval of Orzeyful marks a new chapter for the narcolepsy type 1 community,” said Takeda president and CEO Julie Kim.
