Conditional EU Approval of Muscular Dystrophy Drug Applauded

Pauline Anderson

August 06, 2014

The Muscular Dystrophy Association (MDA), a nonprofit health agency dedicated to finding treatments and cures for neuromuscular diseases, has applauded the decision by the European Union (EU) to conditionally approve ataluren (Translarna, PTC Therapeutics) to treat patients with Duchenne muscular dystrophy (DMD).

"This is a day to remember for the Duchenne community," said Valerie A. Cwik, MD, a neurologist and chief medical and scientific officer, MDA, in a press release. "The approval of ataluren in the EU represents tangible hope that treatments are within reach."

The MDA has been supporting ataluren's development from its early stages, added Dr. Cwik. "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 US and around the world."

DMD is a genetic muscle disease caused by a complete lack of the dystrophin muscle protein. Ataluren is designed to change the way cells read the genetic instructions for the dystrophin protein so that functional dystrophin protein can be produced. It's known as a "stop codon read-through" drug.

Ataluren, which has been an orphan medicinal product since 2005, is the first medication developed specifically for DMD to receive approval in any country.

The EU uses "conditional approval" as a mechanism to allow patients with serious or life-threatening disorders to receive a drug before it has gone through the full approval process. Conditional approval must be renewed yearly and can be revoked if confirmatory studies don't show a drug is safe and effective.

As reported by Medscape Medical News the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) recommended conditional marketing authorization for ataluren after taking a second look at the related data. The CHMP had initially adopted a negative opinion of ataluren, citing insufficient evidence of effectiveness and knowledge of the drug's mechanism of action.

A study of 174 patients with DMD, showed some evidence that ataluren slows down the loss of walking ability, according to the committee. However, as part of the conditional marketing authorization, the company will have to provide comprehensive data on the efficacy of ataluren from an ongoing confirmatory study.

Ataluren is reported to be generally well tolerated, with the most common adverse effects being headache, nausea, and vomiting. A pharmacovigilance plan for ataluren will be implemented as part of the marketing authorization.

The conditional approval is for ambulatory patients aged 5 years and older. The drug is to be available as 125-, 250-, and 1000-mg granules for oral suspension.

Conditional approval in the EU has no direct bearing on approval of a drug in the United States. PTC plans to submit an application for approval of ataluren for DMD in the United States in 2016, after it completes its ongoing, large-scale trial of this drug.

Until now, DMD has been treated with corticosteroids and cardiac medications.

In addition to muscular dystrophy, the MDA funds worldwide research in the areas of amyotrophic lateral sclerosis and other neuromuscular diseases. MDA has supported PTC's development of ataluren through a $1.5 million grant.

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