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A Reminder of Why We Do What We Do for MDA: First Drug for Duchenne Muscular Dystrophy Gets ‘Conditional Approval’ in Europe

CHICAGO, Aug. 4, 2014 — A drug called ataluren (brand name Translarna), in development to treat a subset of patients with Duchenne muscular dystrophy (DMD) has received “conditional approval” in the European Union.

This is the first drug specifically developed for and tested in Duchenne muscular dystrophy to receive approval in any country. PTC Therapeutics, the drug’s developer, announced the approval in an Aug. 4, 2014, press release.

Conditional approval is a designation used in the EU that allows drugs for serious or life-threatening disorders to be made available to patients while results of confirmatory studies are awaited. The approval can be revoked or become a full approval depending on the results of the confirmatory studies. In the meantime, patients can obtain it through their physicians in the 31 European countries (28 countries of the EU and three EU-associate countries).

This is a day to remember for the Duchenne community,” said neurologist Valerie A. Cwik, MDA’s chief medical and scientific officer. “The approval of ataluren in the EU represents tangible hope that treatments are within reach. MDA has been supporting ataluren’s development from its early stages, and we’re eagerly awaiting the results of a large-scale, ongoing trial of this drug that may pave the way for it to become available in the U.S. and around the world.”

Ataluren, developed by PTC Therapeutics of South Plainfield, N.J., acts by changing the way muscle cells interpret genetic information, coaxing them to produce a needed protein called “dystrophin” despite the presence of a mutation in the dystrophin gene. MDA gave PTC $1.5 million for the ataluren’s development in 2005.

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