In another sign that fortunes may be changing for those developing Duchenne muscular dystrophy (DMD) treatments, Prosensa Holding BV announced that it will be pursuing an NDA filing with FDA before year’s end. The news is a positive turnaround for Prosensa after the drug, drisapersen, failed in a Phase III clinical trial and its big pharma partner, GlaxoSmithKline PLC, walked away.
Prosensa Finds Path Forward For Drisapersen In DMD Drug Revival
The Dutch company received positive news for its development programs in the U.S.; an encouraging sign for a disease space that has been flailing in recent months due to unclear clinical endpoints.
More from United States
The agency’s compliance directors also discussed expectations for greater inspection and review efficiencies as part of the 1 October reorganization during a recent FDLI conference.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
Despite GOP criticism of the Part D stabilization program and its budgetary impact, the Trump Administration may decide to keep it in part to avoid blame for a premium increase.
The development of a joint document is not iterative and there is no discussion between the agency and sponsor, according to new guidance seemingly aimed at differentiating the frequent use of joint backgrounders in oncology from the single document developed for the Alzheimer's drug Aduhelm.