New Delhi: Takeda, said on Thursday its narcolepsy pill, the first treatment to target the underlying cause of the rare sleep disorder, is now available in the US 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 treatment is now available by prescription and will be dispensed through Cigna's Accredo Specialty Pharmacy, according to Takeda.
The US Food and Drug Administration had approved Orzeyful 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 US list price for Orzeyful has been set at $16,500 per 60-tablet bottle, across all strengths. At least two analysts said this was below expectations, although in line with current standard-of-care therapies.
The pricing was "somewhat of a surprise" given the enhanced efficacy of the drug class, though the strategy could help expand patient access, said Truist analyst Alex Nackenoff.
About 120,000 people in the US have the disorder, according to Takeda.
"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% of people with this rare disease are not even diagnosed," said Rhonda Pacheco, president of Takeda's US 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.
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