EC ratifies positive opinion for PTC’s Translarna marketing authorisation renewal


The European Commission (EC) has ratified positive opinion for the annual renewal of PTC Therapeutics’ Translarna (ataluren) marketing authorisation for the treatment of nonsense mutation duchenne muscular dystrophy (nmDMD).

The decision is based on the recent renewal recommendation by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

With the authorisation renewal in place, the company will be able to market Translarna for the treatment of nmDMD in ambulatory patients aged five and older within the European Union (EU) member states.

PTC Therapeutics CEO Stuart Peltz said: "As we continue towards our goal of providing Translarna to all who may benefit, we are pleased by the EC ratification of the recent CHMP positive opinion.

"This important regulatory milestone supports the continued growth of our sustainable ex-US business in both the European Union and countries that reference the authorisation.

"Duchenne is a rapidly progressive disease and physicians need access to medicines, like Translarna, that have the potential to slow the devastating progression of this disorder."

“Duchenne is a rapidly progressive disease and physicians need access to medicines, like Translarna, that have the potential to slow the devastating progression of this disorder."

The approval also allows the product to be marketed in European Economic Area members Iceland, Liechtenstein and Norway.

PTC Therapeutics will conduct an additional trial of Translarna in nmDMD as an obligation of the renewal.

In April last year, the UK National Institute for Health and Care Excellence (NICE) recommended Translarna in connection with a managed access agreement (MAA) with NHS England. 

DMD is a progressive muscle disorder caused by the lack of functional dystrophin protein and primarily affects males.