USFDA Approves Takeda's Orzeyful for Rare Narcolepsy Type 1 Sleep Disorder
Bengaluru: The U.S. Food and Drug Administration approved Takeda Pharmaceuticals oral drug for narcolepsy, the health regulator said on Wednesday, backing the first treatment that targets all the symptoms of the rare sleep disorder. Narcolepsy type 1 is a rare sleep disorder caused by the loss of brain cells that produce orexin, a chemical messenger that regulates wakefulness, sleep, and muscle tone.
The condition - affecting about 120,000 people in the United States, according to Takeda - causes people to experience excessive daytime sleepiness, sudden loss of muscle tone, cognitive symptoms and disrupted nighttime sleep.
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Orzeyful, a twice-daily pill, works by directly targeting the loss of orexin signaling that causes the disease.The FDA's approval was based on data from two studies in which patients given Orzeyful showed improvements in their ability to stay awake during the day compared with those given a placebo.Previously, there was no approved medicine for narcolepsy type 1 as a unified disorder, and none acted on the orexin system, the FDA said.The controlled substance classification for Orzeyful is currently under review by the Drug Enforcement Administration, and their ruling is expected within 90 days, the company said.
After the classification is determined, the drug will be made available through a specialty pharmacy to U.S. healthcare providers and patients, Takeda added.
