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.
You may also be interested in...
Pfizer’s DMD Setback With Domagrozumab Shifts Focus To Gene Therapy Approach
With its first AAV gene therapy candidate from Bamboo Therapeutics expected to report data in 2019, Pfizer’s efforts to get in the DMD competition now center on that modality.
Finance Watch: Hua Medicine Raises $117.4m While Evaluating A Hong Kong IPO
Hua, already considering an IPO in Hong Kong for up to $400m, closed concurrent VC rounds that will fund its late-stage diabetes program in China, but it may still need to go public to enter other markets. Also, Heron raised cash for its next commercial product, among other public company financings.
Exondys Revisited? Translarna Brings Efficacy Woes Into US Panel Review
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.