Breakthrough Therapy Designation

In the fifth renewal of PDUFA, section 902 authorized a 'Breakthrough Therapy Designation' to speed regulatory review and (potential) approval of drugs that offer "substantial improvement over existing therapies on 1 or more clinically significant endpoints such as substantial treatment effects observed early in clinical development". 

When a application is submitted, the agency has 60 days to grant 'breakthrough' status or not. If 'breakthrough status' is designated, then the applicant can collaborate closely with the FDA on trial design for the intended patient populations reflecting the most up to date thinking on design.  A quick approval provides potential huge benefits to not only target patient populations but to the company sponsor itself - it could have a tremendous impact on economic state of smaller companies. This could give shot to investors as well with a more predictable pathway and shorter timelines to commercialization.

Thus the door is opened to the FDA for another accelerated approval pathway. The agency is encouraging applications. Said Janet Woodcock, "...We're excited about it.  In fact, although the law is only a few months old, we're already putting it to use. Recently, we identified the first therapy (Vertex's Kalydeco for some CF patients) to this special designation. And it likely won't be long before we have more..."  See Xconomy.

Posted by Bruce Lehr Jan 15th 2013.

Comments

[bigredbruce]

Thanks for the update BDD.

[BioDueDiligence]

VRTX received two breakthrough designations already, and a recent "RPM report" indicated that a third unnamed product has also received breakthrough status already.