FDA provides accelerated approval of Imbruvica (ibrutinib) for marginal zone lymphoma- Janssen Biotech and Pharmacyclics

The FDA has approved Imbruvica (ibrutinib), from Janssen Biotech and Pharmacyclics, for the treatment of patients with marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy. Accelerated approval was granted for this indication based on overall response rate (ORR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

The approval is based on results from the multi-center, open-label single arm, Phase II PCYC-1121 trial assessing the safety and efficacy of IMBRUVICA in 63 patients with MZL who received at least one prior therapy, including all 3 subtypes: mucosa-associated lymphoid tissue (MALT; n=32), nodal MZL (NMZL; n=17), and splenic MZL (SMZL; n=14).

The responses were assessed by an Independent Review Committee (IRC) using criteria adopted from the International Working Group criteria for malignant lymphoma and demonstrated a 46% ORR (95% CI: 33.4-59.1), with 3.2% of patients achieving complete responses (CR) and 42.9% achieving partial responses (PR). Efficacy was observed across all three MZL subtypes (ORR of 46.9%, 41.2%, and 50.0% for MALT, nodal and splenic subtypes, respectively). The median duration of response (DOR) was not reached (range, 16.7-NR), with median follow-up of 19.4 months. The median time to initial response was 4.5 months (range, 2.3-16.4). These data were presented at the 58th Annual American Society of Hematology (ASH) Meeting in December 2016.