European authorities have extended marketing approval for Translarna, PTC Therapeutics Inc’s (NASDAQ:PTCT) treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD), sending its share price rocketing 80%.
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended renewal for ambulatory patients over five years old on the condition there is an additional long-term post-authorisation trial.
“We are pleased with this outcome which took into account all available data for Translarna,” said Stuart Peltz, chief executive.
“Translarna has shown clinically meaningful benefits for patients,” said Eugenio Mercuri, Professor of Pediatric Neurology at the Catholic University in Rome.
“Duchenne is a devastating disease with a progressive loss of function. Maintaining function is of the utmost importance to patients.”
Results from the new study are expected in 2021.
“For boys with Duchenne, every day matters and functional loss cannot be regained. Patients need access to innovative new therapies like Translarna,” added Filippo Buccella, founder of the Italian Parent Project, a patient advocacy group for Duchenne Muscular Dystrophy.
The disease affects boys mainly and is caused by the lack of functional dystrophin, a protein essential for structural stability and muscle development.
Sufferers can lose the ability to walk from the age of ten and often develop fatal lung and heart complications in their teens or early twenties.
Story by ProactiveInvestors