Translarna Renewed for Use by Nonsense Mutation Duchenne MD Patients in Europe

The European Commission (EC) has decided to renew the conditional marketing authorization for Translarna (ataluren) to treat certain nonsense mutation Duchenne muscular dystrophy (nmDMD) patients. PTC Therapeutics can now market Translarna for nmDMD patients who are 5 or older, mobile, and living in the European Union, Iceland, Liechtenstein, and Norway.

The decision follows a renewal recommendation made in November by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

Under the marketing authorization renewal, PTC is required to conduct an 18-month, randomized, placebo-controlled study of Translarna in nmDMD patients. That will be followed by an 18-month, open-label extension period in which all patients will be treatment with Translarna. Results are expected by early 2021.

Most likely, the study will be of similar size to the ACT DMD trial, a multi-center, randomized, double-blind Phase 3 trial (NCT01826487) that evaluated Translarna in 228 patients in 18 countries. Results, reported in late 2015, showed Traslarna’s therapeutic benefits as evaluated based on the North Star Ambulatory Assessment test,  a six-minute walk test, and other timed function assessments.

“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,” Stuart W. Peltz, PhD, chief executive officer at PTC Therapeutics, said in a recent press release. “This important regulatory milestone supports the continued growth of our sustainable ex-US business in both the European Union and countries that reference the authorization. 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.”

Translarna is a small-molecule protein restoration therapy compound designed to allow functional protein to form in patients with genetic conditions caused by a nonsense mutation (a modification in the genetic code that stops the synthesis of an essential protein). The disorder that results from a nonsense mutation is determined by which proteins are being kept from becoming fully functional.

The treatment has not been approved by the U.S. Food and Drug Administration (FDA) for DMD, but it has been designated an Orphan Drug for DMD, cystic fibrosis, and mucopolysaccharidosis I (MPS1).