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Breakthrough Designations Reach 13

May 15, 2013
One of the outcomes of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) was the creation of the “Breakthrough Therapy” designation.  The new law allows FDA to grant a Breakthrough Therapy designation to drugs showing early clinical promise for serious or life-threatening conditions, thereby expediting both the development and the review of these drugs. As of today, 13 designations have been granted according to a search of the BioMedTracker database (see table below).

2013-05-03 breakthroughs3

At the start of 2013, Vertex was the first company to receive the new designation. In fact, Vertex received it for two different Cystic Fibrosis compounds: one for Kalydeco, originally developed for G551D CF patients, and one for VX-809 (listed above as “Lumacraftor”) designed to treat F509del CF patients.

Vertex is one of three biotechs on the above list, the other two being Pharmacyclics and Scioderm. Pharmacyclics has 3 indications listed for the same molecule, Ibrutunib.  Ibrutunib is in Phase III development for various lymphomas, and each indication has been reviewed separately for the designation. Scioderm’s SD-101 for inherited Epidermolysis bullosa, a rare genetic disease, made it to the list soon after Scioderm’s IND filing.

Scioderm’s designation for SD-101 was based on clinical data from a previously conducted Phase II study in a different type of Epidermolysis bullosa. Note that to qualify for the Breakthrough Therapy designation the drug must have preliminary clinical evidence showing substantial improvement on at least one endpoint over available therapy. This may be a high hurdle for some compounds, as 12 applications for the designation have already been rejected.

The other five companies on the list are Big Pharma companies: Abbvie, J&J, Merck, Novartis, and Pfizer. Four of the five Big Pharma companies have Breakthrough Therapies in cancer. That is not too surprising given that more than 50% of the designations are for cancers.

What to expect?

The new designation conveys all of the “fast track” FDA program features, as well as more intensive FDA guidance on development efficiency. Based on Section 902 of FDASIA, we should expect to see the following for the 13 drug programs listed above:

1)      Interactive communication: FDA providing “timely advice” regarding the clinical development and data collection and helping ensure that the “design of the clinical trials is as efficient as practicable”.

2)      Review efficiency: FDA will assign a “cross-disciplinary project lead for the FDA review team”. This person will serve as a scientific liaison between the cross-discipline members of the review team. This review will incorporate senior managers and experienced review staff for the designated drug.

For more on the Breakthrough Therapy designation, here are a couple of useful FDA websites:

Fact sheet


For tracking new designations, here is the BioMedTracker website [subscription required]: