Conditional Okay for Ataluren for Muscular Dystrophy in EU

Megan Brooks

May 23, 2014

Following a second look at the data, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) on May 22 recommended conditional marketing authorization for ataluren (Translarna, PTC Therapeutics) for treatment of Duchenne muscular dystrophy (DMD).

It is to be available as 125-, 250-, and 1000-mg granules for oral suspension, the Committee said.

The indication is for treatment of DMD resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older. "Efficacy has not been demonstrated in non-ambulatory patients," the EMA notes in a statement.

The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing, the agency said, and treatment with ataluren "should only be initiated by specialist physicians with experience in the management of Duchenne/Becker muscular dystrophy."

Ataluren has been an orphan medicinal product since 2005. It is thought to work by enabling the protein-making apparatus in cells to skip over the defect, allowing the cells to produce a functional dystrophin protein, the EMA notes.

CHMP adopted a negative opinion of ataluren in January 2014, citing insufficient evidence of effectiveness and knowledge on the drug's mechanism of action, as reported by Medscape Medical News.

Ataluren was investigated in a study of 174 patients with DMD. "The results showed some evidence of efficacy in slowing down the loss of walking ability in DMD patients. However, as part of the conditional marketing authorization, the company will be required to provide comprehensive data on the efficacy of ataluren from an ongoing confirmatory study," the Committee said May 22.

They note that ataluren is 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.

Detailed recommendations for the use of this product will be described in the summary of product characteristics, which will be published in the European public assessment report and will be available in all official European Union languages after the European Commission grants marketing authorization.


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