Duchenne Muscular Dystrophy: The Race For The $1bn Opportunity

While DMD is proving a difficult area for companies with advanced investigative therapies, several of which have fallen at late-stage development or regulatory hurdles or ignited controversy, the pipeline for new approaches to the disease is dynamic. Analysts estimate the annual market for DMD drugs could reach $1bn, from less than $100m in 2016.

Three products have been approved in the EU or US to treat Duchenne muscular dystrophy: Sarepta Therapeutics Inc.‘s Exondys 51 (eteplirsen) was approved in the US in September 2016; PTC Therapeutics Inc