'Breakthrough' ibrutinib NDA makes rapid arrival at FDA

Ibrutinib notched another milestone in a fast trip toward FDA approval. Johnson & Johnson's ($JNJ) Janssen has delivered the NDA for the closely-watched cancer drug to U.S. regulators, setting the stage for a potential approval on the early side of the time line from the company and partner Pharmacyclics ($PCYC).

The speedy arrival of the marketing application--which covers use of the Bruton's tyrosine kinase inhibitor for three B-cell malignancies--marks another win for drug developers with coveted "Breakthrough Therapy" stamps from the FDA. The FDA's oncology chief, Richard Pazdur, has made clear that such treatments are a top priority, putting them on the inside track to quick approvals.  

The FDA has pinned three "breakthrough" designations for ibrutinib, an indication that regulators have pledged their full support to speeding the experimental drug to patients. The agency is expected to base its decision for approval on impressive mid-stage clinical trial results in patients with chronic lymphocytic leukemia (CLL), the most common form of adult leukemia in the U.S., and other B-cell cancers.

Pharmacyclics has benefited tremendously from the spotlight on its lead drug candidate, with investors betting up its share price, giving the company a market value of more than $6.5 billion. Analysts have based the vast majority of their evaluations of the company on the promise of ibrutinib, which many of them expect to become a major blockbuster with eventual sales of around $5 billion. This would place the drug in a rarefied company with other megahits in oncology such as Gleevec, Rituxan and Avastin. 

"The FDA submission is another important milestone for ibrutinib since we formed our strategic partnership with Pharmacyclics just 18 months ago," Dr. Peter Lebowitz, Janssen's global head of oncology, stated. "Both companies recognize that there is great unmet need among these patient populations, and together in close collaboration with the FDA, as part of its Breakthrough Therapy Designation pathway, we have been able to accelerate the ibrutinib development program for the benefit of patients."

Lebowitz and his partner have asked the FDA to approve the drug for certain patients with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL). Unlike many drug applications that arrive at the FDA's doorstep, J&J's ibrutinib package has prompted analysts to speculate more on "when" rather than "whether" the agency will approve it.

"We predict the drug is on the market before the end of this calendar year," RBC Capital Markets analyst Michael Yee wrote in a note to investors this morning. This timing gives ibrutinib a healthy lead on the market, ahead of rival compounds from Gilead ($GILD) and AbbVie ($ABBV), he noted.

- here's the release

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