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| Pharmacyclics CEO Robert Duggan |
J&J ($JNJ) and Pharmacyclics ($PCYC) nabbed the FDA's new breakthrough designation for their cancer drug ibrutinib, a promising late-stage therapy that will be put in the hands of regulators later this year.
Lawmakers created the breakthrough category to accelerate a select group of programs through a quick review process, possibly shaving a significant amount of time off the slow and expensive development process. The government's idea here is that some drugs that have demonstrated significant promise in offering new hope to patients in early studies should make it to the market without being required to first complete the traditional three-phase development plan.
Ibrutinib fits that bill nicely. The therapy blocks a key enzyme involved in the growth of cancer and helped control the cases of 68% of 116 patients--with a relatively improved safety profile among treatment-resistant patients. The data has driven Pharmacyclics' shares to new heights, making CEO Robert Duggan a paper billionaire in the process. J&J stepped in in late 2011, providing a rich $975 million deal to collaborate on development and marketing.
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| J&J CSO Paul Stoffels |
"We are pleased that the FDA has granted two Breakthrough Therapy Designations for ibrutinib as the designation represents a major leap forward in accelerating drug development timelines," said J&J CSO Paul Stoffels. "We are committed to realizing the full potential of ibrutinib for patients with mantle cell lymphoma, Waldenström's macroglobulinemia, as well as other B-cell malignancies, and will work with Pharmacyclics and the FDA to ensure the clinical development program for ibrutinib continues to move forward as quickly as possible."
Ibrutinib follows two of Vertex's ($VRTX) cystic fibrosis drugs--Kalydeco and VX-809, being studied as a combination treatment--down the new breakthrough path at the FDA. All three are being managed by well-known development teams and earned a high profile for early promise. And the industry will be paying close attention to the FDA's announcements on future breakthrough designations, looking for more signs of what the agency is looking for.
The FDA's Janet Woodcock stirred considerable interest yesterday with her remark that it will be possible to win an approval based on expanded Phase I data. But given its cautious history on the approval front, it's likely that they will stick with proven developers who have a solid reputation, working in areas where the biology of the disease is well known, especially as they move up the R&D stream. As the FDA proved recently with its rejection of the diabetes drug Tresiba, it won't hesitate to require major new studies before green-lighting any drug headed for a mass market.
- here's the press release
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