Pilot program that gives agency early look at pivotal data has forced sponsors to change how they go about preparing supplemental applications; potential expansion to new molecular entities would bring new challenges, including need to align manufacturing and clinical site inspections with expedited review timeline.
The US FDA’s Real-Time Oncology Review (RTOR) pilot program has required participating sponsors to change how they prepare and submit supplemental applications, but these challenges have come with some big payoffs, including greater certainty about application acceptance and review times that can be counted in days and weeks, as opposed to months.
While RTOR currently is being piloted for only the most straightforward supplemental applications, future expansion to more complex supplements or...
The European Medicines Agency has recommended against pan-EU marketing authorization of Roche/Sarpeta’s gene therapy Elevidys for the treatment of Duchenne muscular dystrophy, despite the product securing approvals in the US and other markets.
After three months on the job, FDA Commissioner Martin Makary is sounding much more effusive about the quality of the agency staff. Better late than never?
Replimune's RP1 oncolytic immunotherapy became the third regenerative medicine to receive an FDA complete response letter this month as the agency appears to re-visit previous agreements about accelerated approval.
