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.
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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Menarini's Asia-Pacific CEO talks to Scrip about how the Italian group is tailoring its portfolio and pricing approach to local markets making strong strides in primary care and consumer health, while also charting a distinct path in rare diseases and oncology. The China market is also a key focus including as a potential source of innovation as is partnering, where the group has a rich history.
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The small interference RNA therapeutic can be used by patients regardless of inhibitor status.
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Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.