A ‘Transformative’ Narcolepsy Drug Opens New Opportunities in Neuroscience

Many people have a cartoonish idea of narcolepsy, perhaps picturing someone falling asleep standing up.

What people don’t understand is that sleepiness can infiltrate every aspect of daily life, said Julie Flygare, 42, who was diagnosed with the sleep disorder in 2007, during her second year of law school. It interferes with memory, attention and weighs down everyday existence like a “heaviness on the skull,” she said.

While taking her current medications, Ms. Flygare has four to six precious hours of wakefulness a day, and still needs a nap. Positive feelings, like, say, the joy of hitting a volley in tennis, trigger episodes of cataplexy; her muscles go slack and her grip on the racket loosens. She, like many other patients, struggled for years to be correctly diagnosed and to find the right combination of stimulants or other drugs to ease her symptoms.

Since then, Ms. Flygare has been waiting, and pushing for, a drug aimed not just at alleviating sleepiness, but at replacing a crucial missing brain chemical. Last month, she saw it happen. A first-of-its-kind drug for the sleep disorder narcolepsy was approved by the F.D.A., offering a new treatment for people who live with the debilitating fog of sleepiness.

The new drug developed by Takeda Pharmaceutical Co., called Orzeyful, marks a long-sought success in the quest to mimic an essential brain peptide called orexin, which is missing in people with type 1 narcolepsy. About 120,000 people in the United States suffer from this type of narcolepsy, which causes pervasive sleepiness and episodes of cataplexy. Other companies, including Alkermes and Eli Lilly, are chasing close behind.

To test narcolepsy treatments, doctors use a “maintenance of wakefulness test” in which a person sits in a dim room for 40 minutes.