WASHINGTON, D.C. — The U.S. Food and Drug Administration approved Takeda Pharmaceuticals America’s Orzeyful tablets for adults with narcolepsy type 1, making it the first treatment cleared to address the disorder as a whole by directly targeting the loss of orexin signaling that underlies the disease.
Orzeyful, or oveporexton, is taken twice daily and activates the same brain receptor normally stimulated by orexin, a chemical messenger involved in wakefulness, sleep and muscle tone.
Narcolepsy type 1 is a rare, lifelong neuropsychiatric sleep disorder affecting an estimated 1 in 2,000 people in the U.S. It is caused by the loss of brain cells that produce orexin.
The condition can cause excessive daytime sleepiness, cataplexy, sleep paralysis, hallucinations around sleep transitions and disrupted nighttime sleep.
“For too long, people with narcolepsy type 1 have had to manage a complex, lifelong neuropsychiatric condition with treatments that only address pieces of it,” Tiffany R. Farchione, director of the FDA’s Division of Psychiatry, said.
Farchione characterized Orzeyful as the first approved medicine to act on the disease’s underlying biology rather than only individual symptoms.
The FDA based the approval on two randomized, double-blind, placebo-controlled studies involving 273 adults with narcolepsy type 1.
Across the 12-week trials, patients receiving the 2 mg dose showed greater improvement in daytime wakefulness than those receiving placebo, according to the agency.
Patients also reported less daytime sleepiness, fewer cataplexy episodes and improvement in other symptoms, including sleep paralysis, hallucinations and disrupted nighttime sleep.
The most common side effects were insomnia, increased urinary frequency, urinary urgency and increased saliva production. Few participants discontinued treatment because of adverse effects, according to the FDA.
Orzeyful should not be used with strong CYP3A inhibitors, and its safety and effectiveness have not been established in patients younger than 18.
The drug has been recommended for scheduling under the Controlled Substances Act and cannot be legally marketed until the Drug Enforcement Administration issues its scheduling decision.
Orzeyful previously received Breakthrough Therapy Designation and Priority Review from the FDA, programs intended to accelerate development and review of therapies that may offer meaningful improvements for serious conditions.
The approval was granted to Takeda Pharmaceuticals America Inc.
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