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European approval beckons for muscular dystrophy drug

Child in wheelchair (Aldegonde Le Compte/

Ataluren will be available for children aged five years and older whose disease is caused by defects in the dystrophin gene

The first drug of its kind to treat a particular form of Duchenne muscular dystrophy is being recommended for use in the European Union by the European Medicines Agency (EMA).

Ataluren encourages the cell to ignore ‘nonsense mutations’ that gradually cause weakness and loss of muscle function. It will be available for children aged five years and older whose disease is caused by defects in the dystrophin gene. This disorder is estimated to affect around 200 children and young people in the UK.

The EMA’s Committee for Medicinal Products for Human Use initially decided not to approve the drug in January, but reappraised the data following a request from the medicine’s manufacturer, PTC Therapeutics.

The committee decided last Friday (23 May 2014) that there is enough evidence to recommend the drug on the condition that the manufacturer provides it with ongoing results from its clinical trials.

The decision is described as an “important milestone for drug development programmes in Duchenne muscular dystrophy” by Volker Stroub, a neuromuscular researcher at Newcastle University, UK.

Conditional marketing authorisation is an early access mechanism that allows the EMA to recommend authorisation for medicines that address an unmet medical need, even if comprehensive clinical data are not yet available.

The European Commission must approve the recommendation before the drug can be made available in member states.

Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2014.11138704

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