Orzeyful, an oral medicine created by Takeda Pharmaceutical Company in Tokyo, addresses a full range of narcolepsy symptoms by activating the part of the brain affected by the disorder.
“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,” said Tiffany Farchione, director of the division of psychiatry at the FDA’s Center for Drug Evaluation and Research.
Until now, treatments have targeted only symptom relief. Orzeyful is the first medicine that impacts the underlying biology of the disease, Farchione said.
“The FDA approval of Orzeyful marks a new chapter for the narcolepsy type 1 community, as we introduce an entirely new class of medicine that will potentially redefine how this disease is managed and how people feel on treatment,” said Takeda President and CEO Julie Kim.
“We are proud to have discovered and developed this orexin treatment innovation and will bring it to adults living with [narcolepsy] as quickly as possible,” Kim said.
Narcolepsy type 1 is a rare, chronic neurological disease. Type 1 involves “cataplexy,” an episode triggered by a strong emotion. The emotion causes a sudden loss of muscle tone and can result in a full-body collapse. Type 2 narcolepsy doesn’t involve cataplexy, according to the Narcolepsy Network.
The National Institute of Neurological Disorders and Stroke estimates that 120,000 to 200,000 people in the United States live with narcolepsy. People with the condition may feel rested after waking for the day but then feel sleepy through the day, according to the National Institutes of Health (NIH).
Once narcolepsy develops, a person with the disorder will have it for life, the NIH reported.
Narcolepsy type 1 is caused by the loss of brain cells that produce orexin, a chemical messenger that regulates wakefulness, sleep, and muscle tone, according to the FDA. Without orexin, the brain can’t stay alert or control the boundary between sleep and being awake.
As a result, people with the disorder have a variety of disabling symptoms, including excessive daytime sleepiness, cataplexy, sleep paralysis, hallucinations, and disrupted nighttime sleep.
Orzeyful works to directly activate the brain receptor that the body’s orexin would stimulate if it weren’t missing the signal, according to the FDA. The medicine is taken as an oral tablet twice a day.
Orzeyful was evaluated in two randomized, double-blind, placebo-controlled 12-week studies with 273 adults with narcolepsy type 1. Patients in both studies taking Orzeyful 2 mg showed improvements in their ability to stay awake compared with those taking placebos.
They also reported less daytime sleepiness and fewer cataplexy episodes, along with other meaningful improvements, the FDA reported.
The medication’s approval opens up new therapeutic possibilities for other conditions, according to Takeda’s president of research and development Andy Plump.
“Orzeyful is just the beginning,” Plump said. “With orexin emerging as a powerful therapeutic pathway, Takeda is unlocking the potential of this new class of medicine for patients across a range of disorders.”
Orzeyful is still under review by the U.S. Drug Enforcement Administration (DEA) for its controlled substance classification, which is expected within 90 days.
