Pharmacyclics and Janssen Research & Development announced that the FDA has granted Breakthrough Therapy Designation for the investigational oral drug ibrutinib as monotherapy in patients with relapsed or refractory mantle cell lymphoma (MCL), and in patients with Waldenstrom’s macroglobulinemia (WM). Ibrutinib is designed to specifically target and selectively inhibit an enzyme called Bruton’s tyrosine kinase (BTK). BTK is a key mediator of at least three critical B-cell pro-survival mechanisms occurring in parallel-regulating apoptosis, adhesion, and cell migration and homing.
The Breakthrough Therapy Designation submission was based on data from clinical and pre-clinical studies. Pharmacyclics and Janssen have designed a comprehensive development program of ibrutinib in MCL which includes two Phase 2 and two Phase 3 studies.