FDA approves Imbruvica for CLL

FDA granted accelerated approval to Imbruvica ibrutinib from Pharmacyclics Inc. (NASDAQ:PCYC) and partner Johnson & Johnson (NYSE:JNJ) to treat chronic lymphocytic leukemia (CLL) in patients who have received at least one prior therapy. The approval -- which triggers a $60 million milestone payment to Pharmacyclics from J&J -- was based on data from a Phase Ib/II trial showing the drug led to an overall response rate (ORR) of 58.3% in 48 patients with relapsed or refractory CLL or small cell lymphocytic lymphoma (SLL). Pharmacyclics had been seeking approval for both CLL and SLL, but according to the company, FDA said data for the three SLL patients in the trial was "too small to make a conclusion about efficacy." Imbruvica already has accelerated approval from FDA for mantle cell lymphoma (MCL) in patients who have received at least one prior therapy. Pharmacyclics and J&J have not yet broken out sales of the drug, which the partners launched in November (see BioCentury Extra, Nov. 13, 2013).

Imbruvica has breakthrough therapy designation from FDA for MCL and for CLL or SLL in patients with chromosome 17p deletion, as well as for Waldenstrom's macroglobulinemia. The compound is a Bruton's tyrosine kinase (Btk) inhibitor that covalently binds to cysteine residue 481. In October, J&J submitted an MAA to EMA for ibrutinib to treat MCL and CLL or SLL. ...