FDA approves Pharmacyclics and Janssen’ Imbruvica to treat Waldenstrom’s macroglobulinemia

Imbruvica is an once-daily oral therapy that inhibits a protein called Bruton’s tyrosine kinase (BTK). It also secured breakthrough therapy designation for WM from the FDA and is being jointly developed and commercialized by Pharmacyclics and Janssen Biotech.

The approval was based on data from a multi-center, Phase II trial that evaluated the efficacy and tolerability of Imbruvica 420mg in 63 patients with previously treated WM.

In the trial, Imbruvica showed a response rate of 62% as assessed by an Independent Review Committee.

International Waldenstros Macroglobulinemia Foundation president Carl Harrington said: "The FDA’s approval of Imbruvica for Waldenstrom’s macroglobulinemia marks a significant milestone for patients living with this rare disease and has the potential to positively impact the lives of a number of patients."

Pharmacyclics Hematology head Thorsten Graef said: "Imbruvica has demonstrated its effectiveness in patients living with Waldenstrom’s macroglobulinemia who, until now, did not have an FDA-approved standard-of-care therapy.

"This approval is a proud moment for our Pharmacyclics and Janssen teams and would not have been possible without the dedication and support of our patients, clinical investigators and the FDA’s recognition of Imbruvica as a breakthrough therapy for these patients."

Currently, Imbruvica is approved to treat patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy, CLL patients with del 17p and patients with Waldenstrom’s macroglobulinemia.

The drug is also approved to treat patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.