A Japanese pharma company has developed a drug that treats the root cause of a leading form of narcolepsy, and is celebrating as it becomes the first of its kind to receive American FDA approval.
Narcolepsy type-1 afflicts 120,000 people in the US, and is characterized by sudden bouts of intense daytime drowsiness, disrupted nighttime sleep, and cataplexy, or sudden muscle weakness while awake
Traditionally treated by simulants to stay awake, or nervous system depressants to dampen cataplexy, this new drug mimics the body’s production of orexin, a compound that helps regulate wakefulness that is disrupted in patients with narcolepsy.
Called Orzeyful, it’s been developed by Takeda, and Tiffany Farchione, MD, Director of the FDA’s Division of Psychiatry, celebrated its approval in an August 5th statement.
“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 Farchione. “This new drug is the first medicine that impacts the underlying biology of the disease, treating narcolepsy type 1 as a whole.”
Its developers likewise celebrated the milestone.
“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,” says Takeda president and CEO Julie Kim in a separate statement.
The final stage trials that paved the way for approval demonstrated safety and efficacy in 273 patients. Compared to placebo, those who took the twice-daily pill for 12 weeks were able to stay awake longer during the day and rest better at night.
“It’s the best thing that has ever happened to me,” Tyler Chapman, who participated in one of the clinical trials, told Nature’s in-house press publication. “My quality of life is so much better.”
NEW THERAPEUTICS: New Kind of Antibiotic Is First in 30 Years to Treat UTIs That Keep Coming Back
There were side effects, including of a severity that saw some participants removed from the trial. The most common serious side effects were increased frequency and urgency to urinate, and—ironically—insomnia. Nevertheless the side effect occurrence was considered low.
Orexin, the chemical signaling molecule produced by brain cells which die in narcolepsy type-1, has been linked to other neurological conditions like ADHD, and even Parkinson’s, and with Orzeyful’s FDA approval comes the potential for future research into its utility as a neuro-modulator.
ALSO CHECK OUT: New Drug Could Change Lives of Kids with Resistant Epilepsy, Patient Trials Concluded
“Orzeyful is just the beginning. 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,” says Andy Plump, the company’s president of research and development, in also the statement.
WAKE UP YOUR FRIENDS To This Great New Medication By SHARING This Story…
