FDA’s DMD Decisions: Glass Two-Thirds Full?
Executive Summary
The outcome of an US advisory committee review of PTC’s ataluren was not surprising. That is bad news for PTC’s prospects of launching the drug in the near term – but given the recent history of the DMD class, it is probably good news for biopharma companies overall as a sign that FDA's neurology group is moving forward effectively.
You may also be interested in...
PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?
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.
Partisan Politics Returns To US FDA Congressional Oversight
The US FDA has stood out as an agency that tends to draw broad bipartisan support amid a generally rancorous and divided Congress. A House hearing, however, may be a sign that those days are over.
Medicare Reaffirms Faith In Formulary Review Process Ahead Of Part D Changes
Dramatic transformation in the US Medicare Part D benefit design does not require any changes to CMS’ overall approach to reviewing formulary submissions from private drug plan sponsors, the agency says in its final guidance implementing the design changes for 2025.