In September, the U.S. Food and Drug Administration formally approved elamipretide as a treatment for Barth syndrome under an expedited process for rare diseases. The achievement is the realization of a two-decade-long drug discovery journey that began with a groundbreaking discovery by pharmacologist Dr. Hazel Szeto, M.D., Ph.D. ’77, at Weill Cornell Medicine, who discovered the compound that became elamipretide and was the scientific founder of a biotechnology company to commercialize it.
Elamipretide is the first compound discovered at Weill Cornell Medicine to attain FDA approval; it will be available on the market starting this month under the brand name Forzinity to improve muscle strength in adults and children weighing at least 30kg with Barth syndrome.
“This is a monumental accomplishment for Dr. Szeto and Weill Cornell Medicine,” said Dr. Lisa Placanica, senior managing director of the Center for Technology Licensing at Weill Cornell Medicine. “Navigating the long journey through the drug discovery pathway and reaching this incredible milestone required the commitment of numerous institutional, investor, patient advocate and commercial stakeholders. We extend our profound congratulations to Dr. Szeto for her tireless efforts.”
“I went to medical school, but I chose not to practice,” Dr. Szeto said. “And I figured, one day, I would invent a drug that would then be able to reach far more patients than I would be able to singlehandedly treat in an office. So, this is my dream come true.”
