Analysts score the winners in the FDA's new 'breakthrough' category

A year after the FDA began to roll out the first of its breakthrough therapy designations, the analysts at Leerink Swann have concluded that while the jury is still out on whether or not a BTD will mean a significantly shortened path to the marketplace, it's proven to be a big influence on investors watching the industry.

So far, says Leerink, 37 drugs have won BTD designations, representing a big commitment for an agency which says it is determined to provide easy access for investigators on the VIP list. The breakthrough designation also appears to be playing a particularly important role in cancer R&D.

But even as more and more developers are rushing to the FDA applying for breakthrough designations, the agency is proving more reluctant when it comes to providing the distinction. The rejection rate doubled in the latter part of 2013.

"By therapeutic area, cancer compounds represent the largest group (12, or 41%), followed by agents for orphan diseases (9, or 31%), and anti-infectives (6, or 21%)," notes Leerink. "For the 29 granted BTDs that have been publicly announced, Johnson & Johnson ($JNJ) leads all companies with four, although three of these are for Imbruvica (jointly developed with Pharmacyclics). GlaxoSmithKline ($GSK) and Novartis ($NVS) each has 3 BTDs granted for 3 separate agents. By type of compounds, clearly being first in class is not a requirement, and "me-better" agents such as CD20 antibodies (Gazyva and Arzerra) and ALK inhibitors (LDK378 and alectinib) are definitely eligible. By nature of data, efficacy signals from both single arm studies (such as tumor regression) and Phase II comparative studies have been the basis for BTD."

FDA cancer drug chief Richard Pazdur made a highly public pledge at ASCO last summer to speed up the approval times of important new cancer remedies. While the BTD stamp may have shortened the timelines for the three breakthrough therapies that were approved last year--Sovaldi, Imbruvica and Gazyva--FierceBiotech notes that we're still quite a ways from seeing a radical change on timelines.

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