Takeda 4502.T said on Thursday its narcolepsy pill, the first treatment to target the underlying cause of the rare sleep disorder, is now available in the U.S. after a Drug Enforcement Administration decision removed the final regulatory hurdle for its rollout.
The DEA has classified Orzeyful as a Schedule IV controlled substance, the Japanese drugmaker said, a category considered to have a low potential for abuse and dependence. Schedule II drugs face stricter controls and Schedule V drugs the least.
The U.S. Food and Drug Administration had approved the treatment in August for narcolepsy type 1, a rare disorder caused by the loss of brain cells that produce orexin, a hormone that affects the brain’s control over sleep and wake cycles.
Instead of just treating the symptoms, Orzeyful, a twice-daily pill, targets the loss of orexin signaling that causes the disorder, which is characterized by excessive daytime sleepiness, sudden loss of muscle tone, cognitive symptoms and disrupted nighttime sleep.
The condition affects about 120,000 people in the U.S., according to the company.
Orzeyful is now available by prescription and will be dispensed through Cigna’s CI.N Accredo Specialty Pharmacy, Takeda said.
“Our initial launch efforts will be on adults who are cycling through other treatments or taking multiple medications and still experience breakthrough symptoms. And there is a broader opportunity given 50 per cent of people with this rare disease are not even diagnosed,” said Rhonda Pacheco, president of Takeda’s U.S. business unit.
In studies, the drug showed improvements in patients’ ability to stay awake during the day compared with placebo, and significantly reduced instances of cataplexy, a sudden muscle weakness triggered by strong emotions.
Reporting by Siddhi Mahatole and Sriparna Roy in Bengaluru; Editing by Shreya Biswas, Caroline Humer and Jonathan Ananda
