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.
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...
A court order encompassing funding, drug pricing, clinical trials and overall policy implementation aspects is expected to bring about a paradigm shift in the treatment of rare diseases in India. Sarepta, Roche and Sanofi are among the key players that have been part of pricing discussions.
Merck & Co. won European Commission approval for Winrevair on top of other medicines for pulmonary arterial hypertension and expects an initial EU launch in Germany.
The company, in its new profitable era, is bringing in new leadership, with Amgen VP of medical affairs for rare diseases Greg Friberg and recently retired Roche Partnering head James Sabry.
Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.
Mounjaro, Eliquis, and Darzalex buck the Q1 trend to make gains
