CAMBRIDGE, Mass. — The U.S. Food and Drug Administration approved Takeda’s ORZEYFUL for adults with narcolepsy type 1, making it the first approved treatment designed to address the underlying orexin deficiency associated with the disorder.
ORZEYFUL, also known as oveporexton, is an oral orexin receptor 2 agonist. Takeda said it expects to make the drug available in the United States after completion of the Drug Enforcement Administration’s scheduling process.
Narcolepsy type 1 is a rare, chronic neurological condition caused by the loss of orexin, a chemical involved in regulating wakefulness and sleep. The disorder can cause excessive daytime sleepiness, sudden loss of muscle tone known as cataplexy, cognitive symptoms and disrupted nighttime sleep.
Takeda estimates that the condition affects about 120,000 people in the United States.
“The FDA approval of ORZEYFUL marks a new chapter for the narcolepsy type 1 community, as we introduce an entirely new class of medicine,” Takeda President and CEO Julie Kim said.
The approval was supported by results from the global Phase 3 FirstLight and RadiantLight trials.
Takeda said patients treated with oveporexton experienced statistically significant improvements in excessive daytime sleepiness, cataplexy and health-related quality of life compared with patients who received a placebo.
The drug was generally well tolerated across the clinical program. The most commonly reported side effects included insomnia, urinary urgency, frequent urination and excessive saliva.
Existing treatments for narcolepsy type 1 have generally focused on managing individual symptoms. ORZEYFUL is designed to restore signaling through the orexin pathway and address a broader range of symptoms associated with the disease.
“Until now, people have managed narcolepsy type 1 with treatments that target symptom relief,” said Emmanuel Mignot, M.D., Ph.D., principal U.S. investigator in the Phase 3 program. “As the first and only approved orexin therapy to treat the broad spectrum of the disease, ORZEYFUL can enable a different kind of conversation in the doctor’s office about treatment options.”
Takeda said the approval represents the first commercial application of its orexin drug-development program. The company is also studying the pathway as a potential treatment target for other disorders.
