Duchenne Muscular Dystrophy: The Race For The $1bn Opportunity
With three products now approved in Europe and/or the US for Duchenne Muscular Dystrophy, an FDA decision pending on PCT Therapeutics' ataluren and multiple companies exploring different approaches in preclinical and clinical studies, Scrip provides an overview of the development landscape and commercial results to date.
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FDA has twice refused to file PTC Therapeutics' NDA for the Duchenne muscular dystrophy treatment because of 'clearly and convincingly negative' pivotal trials and questionable post hoc analyses; sponsor argues ataluren should be afforded the same consideration and flexibility given to Sarepta's DMD drug eteplirsen, which received a controversial accelerated approval.