EMA Body Recommends Translarna as First In-Class Treatment of Duchenne Muscular Dystrophy

Pharmaceutical firm PTC Therapeutics has received conditional marketing authorization in Europe for Translarna, the firm’s experimental therapy to treat Duchenne muscular dystrophy (DMD) in patients five years and older. 

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended granting a conditional marketing authorization for the therapy, EMA said May 23. The CHMP opinion now goes to the European Commission, which will issue a decision on an EU-wide marketing authorization.

DMD is a genetic disease that gradually causes the weakness and loss of muscle functions. Translarna (ataluren) would treat a small sub-group of patients affected by certain defects known as nonsense mutations.

The EMA noted that currently there are no approved therapies for this life-threatening condition, and that management of the disease is based on prevention and managing complications.

PTC Therapeutics said it expects to have all patients enrolled in a global Phase III clinical trial for the drug by mid-2014. The outcome of this trial is critical for achieving full approval in the EU as well as the U.S., the company said May 23.

In January 2014, CHMP originally took a negative stance towards Translarna but quickly changed its tune due to requests from applicants and a positive re-assessment of data. The Telegraph’s Medical Editor Rebecca Smith described the agency’s move in a May 23 article as “a historic U-turn by medicines regulators.”  — Harry Morrison

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