Full FDA approval for Imbruvica in CLL

FDA granted full approval to Imbruvica ibrutinib from Pharmacyclics Inc. (NASDAQ:PCYC) and Johnson & Johnson (NYSE:JNJ) as first-line therapy for chronic lymphocytic leukemia (CLL) in patients with 17p deletion and to treat CLL in patients who have received at least one prior therapy. FDA granted accelerated approval to the drug for the latter indication in February. The full approvals -- which trigger $60 million in milestone payments to Pharmacyclics from J&J -- came over two months ahead of the Oct. 7 PDUFA date. Pharmacyclics said Imbruvica is the first drug approved specifically for the 17p deletion population, and estimated that 3-7% of de novo patients have 17p deletion at diagnosis and an additional 30-40% of patients may have acquired the deletion during the course of disease. The Bruton's tyrosine kinase (Btk) inhibitor that covalently binds to cysteine residue 481 is already available in the U.S. at a wholesale acquisition cost (WAC) of $8,200 for a 30-day supply. The median treatment duration was 8.6 months in the Phase III RESONATE (PCYC-1112-CA) trial.

The full approvals were based on data from RESONATE, which was stopped early after an interim analysis in January showed that Imbruvica met the primary endpoint of improving progression-free survival (PFS) vs. IV Arzerra ofatumumab from GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) and Genmab A/S (CSE:GEN; OTCBB:GMXAY) in patients with relapsed or refractory CLL or small lymphocytic leukemia (SLL). Pharmacyclics had been seeking full approval for both CLL and SLL, but said it agreed with FDA to gather a larger SLL sample since the SLL patient population in RESONATE consisted of 18 patients (see BioCentury, Jan. 13). ...