Brazilian Scientist Sees 25‑Year Journey Lead to a Promising Narcolepsy Drug

A Brazilian researcher who uncovered the genetic cause of narcolepsy more than two decades ago is finally seeing his work advance toward a potential treatment. Mário Pedrazzoli, 61, a biologist and professor at the University of São Paulo, identified the genetic mechanism behind the disorder during his postdoctoral studies at Stanford University. The findings were published in Nature Medicine in 2000.

At the time, Pedrazzoli believed a therapy could reach the market within a few years. “Science rarely moves at the pace we imagine,” he said. “But persistence is part of the process.”

Twenty‑five years later, researchers announced positive results from the final phase of clinical trials for a new drug, ovaporexton, developed by the Japanese pharmaceutical company Takeda. The medication now awaits review by the U.S. Food and Drug Administration.

Experts say the therapy could mark a turning point. “A targeted, continuous‑use treatment in pill form represents a major advance for patients,” said Dr. Elaine Roberts, a sleep‑medicine specialist in Boston. “Narcolepsy affects millions worldwide, and current options are limited.”

Narcolepsy is a neurological disorder that disrupts the brain’s regulation of sleep, causing excessive daytime drowsiness and sudden episodes of sleep. An estimated 3 million people live with the condition globally.

For Pedrazzoli, the progress is both scientific and personal. “Seeing a discovery leave the lab and move toward helping real people is the greatest reward a researcher can hope for,” he said.

Mário Pedrazzoli, biologist and professor at the University of São Paulo