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
• By
Pharma executives and investors are waiting with bated breath to find out if President Trump will include drugs in a new round of tariffs to be announced on 2 April.
• By
One CDRH employee said the cuts already are having a major effect on morale.
• By
Mass FDA layoffs on 1 April were designed to spare product reviewers, but still touched many who are critical to the application review process or drug development, which could mean fewer treatments are brought to the US market first.
• By
Experts say PBMs should become familiar with transparency rules and prepare to release data, although more legal wrangling could occur.
More from North America
• By
Optum Rx, one of the largest PBMs in the US, declared it was the “first comprehensive, transparent pharmacy services company” after announcing several consumer-friendly changes.
• By
FDA Commissioner Martin Makary was sworn in Friday and knew of the plan that ultimately lead to CBER director Peter Marks' resignation on 28 March.
• By
In his resignation letter, the CBER director said he was willing to work with HHS Secretary Robert F. Kennedy Jr. to address concerns about vaccine safety, but "it has become clear that truth and transparency are not desired by the secretary."