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FDA's Breakthrough Therapy Designation More Bling Than Bite

By Brian Orelli, PhD - Apr 10, 2013 at 9:43AM

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Companies say, "the implications of Breakthrough Therapy Designation cannot be determined at this time."

If one "Breakthrough Therapy Designation" is good, three must be great.

That is, if we knew what the designation was really worth.

Johnson & Johnson (JNJ 1.13%) announced on Monday that it had received a third Breakthrough Therapy Designation for the blood cancer drug ibrutinib that it's developing with Pharmacyclics (NASDAQ: PCYC). The designation covers patients with chronic lymphocytic leukemia or small lymphocytic lymphoma who have a deletion of the short arm of chromosome 17.

In February, the Food and Drug Administration gave the drug the designation for patients with relapsed or refractory mantle cell lymphoma that failed prior therapy and for patients with Waldenstrom's macroglobulinemia, a type of cancer affecting B cells.

The Breakthrough Therapy Designation was part of the last year's FDA Safety and Innovation Act, which is supposed to speed up development review time -- potentially requiring less data for approval -- for drugs that treat serious diseases where there isn't a treatment or where the product in development is a vast improvement over current options.

But Congress, in its infinite wisdom, left exactly how to implement the benefits up to the FDA. That was probably a good idea in the long run since the FDA knows what drugmakers need, but the agency isn't particularly quick in establishing rules.

Companies are left making announcements about a designation that they don't know exactly how they'll benefit from. "The implications of Breakthrough Therapy Designation cannot be determined at this time," read the press release by Johnson & Johnson's subsidiary Janssen Research & Development.

Vertex Pharmaceuticals (VRTX 1.96%) had the exact same wording -- maybe their flack went to the same law school -- in the announcement that the FDA had given Breakthrough Therapy Designation to its cystic fibrosis drug Kalydeco as both a monotherapy and in combination with VX-809.

The biotech added that it's working with the FDA and other regulatory authorities to figure out exactly how it'll affect the development. It sure doesn't look like the designation will help reduce the size of the clinical trials required for approval. Vertex subsequently announced that it's started two 500-patient phase 3 trials testing VX-809 with Kalydeco in cystic fibrosis patients with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

The designation might not have much benefit, but don't ignore the drugs involved; both Vertex's combination treatment and ibrutinib have a lot of promise. Just this week, promising data from a clinical trial testing ibrutinib in hart-to-treat chronic lymphocytic leukemia patients was presented at the American Association for Cancer Research annual meeting.

At this point, the Breakthrough Therapy Designation is like wearing a broken luxury watch; it's a nice status symbol, but it isn't particularly useful. Some will argue that it says that the FDA thinks the drug has promise, but that should be obvious to most investors anyway. The drugs still have to prove their worth in clinical trials to get approved.

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