PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?
• By Michael McCaughan
US FDA appears willing to consider accelerated approval for PTC's Duchenne Muscular Dystrophy therapy ataluren if company is able to provide compelling evidence of increased levels of dystrophin in boys with nonsense-mutation DMD. That is quite a reversal from the agency’s view heading into PTC’s appeal.
Precedent or Precipice? The US FDA might be headed into a beautiful landscape – or towards a steep, rocky cliff. • Source: Tony Hisgett
Is history repeating itself with another potential therapy to treat Duchenne Muscular Dystrophy?
While treatments for scleroderma are in development, none are approved for the condition that is associated with high morbidity and mortality. The European Medicines Agency says guidance on how to design development programs for the disorder is needed.
As Chief Medical and Scientific Officer, Vinay Prasad will advise the FDA Commissioner on cross-cutting issues, provide strategic leadership and be a public face of the agency.
US FDA Commissioner Martin Makary will establish "national priorities" used to select sponsors for the pilot of “tumor board-style” drug reviews that would start before Phase III is completed.
