PTC Therapeutics Surges 88% On European Marketing Authorization

EMA's Translarna Recommendation

Translarna is a therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older.

The announcement is a welcome development for PTC's investors following a denial letter from the U.S. Food and Drug Administration (FDA) on October 17.

As part of the marketing authorization, PTC Therapeutics will conduct a new 18-month randomized, placebo-controlled study in patients suffering from nmDMD.

Results are expected to be available in the first quarter of 2021.

Expert Voices

"We are pleased with this outcome which took into account all available data for Translarna," said Stuart W. Peltz, Ph.D., PTC Therapeutics CEO. "This decision reflects the benefit that Translarna is having for patients suffering from nonsense mutation Duchenne muscular dystrophy."

"Translarna has shown clinically meaningful benefits for patients," said Eugenio Mercuri, M.D., professor of pediatric neurology at the Catholic University, Rome, Italy. "Duchenne is a devastating disease with a progressive loss of function. Maintaining function is of the utmost importance to patients."

"The consistency of Translarna's benefit shown across key endpoints is impressive for a dystrophin replacement therapy," said Craig McDonald, M.D., professor of pediatrics and chair of the department of physical medicine & rehabilitation at University of California. "I am encouraged for the DMD community by the CHMP's recommendation."

"For boys with Duchenne, every day matters and functional loss cannot be regained. Patients need access to innovative new therapies like Translarna," stated Filippo Buccella, founder of the Italian Parent Project, a patient advocacy group for Duchenne Muscular Dystrophy.