Janssen, Pharmacyclics File sNDA For Imbruvica In WM

Janssen Research & Development reported that its partner Pharmacyclics has filed a supplemental New Drug Application (sNDA) with the U.S. Food and Drug Administration (FDA) for Imbruvica as treatment for a rare type of B-cell lymphoma known as Waldenstrom's macroglobulinemia (WM).

Imbruvica (ibrutinib) specifically targets and inhibits the enzyme Bruton's tyrosine kinase (BTK), which has been shown to help direct B-cells to lymphoid tissues and access a microenvironment that enables them to survive. The drug is approved in three indications, including treatment of chronic lymphocytic leukemia (CLL). In April, Pharmacyclics filed a separate sNDA for the drug in CLL and mantle cell lymphoma (MCL). Earlier this month, Janssen-Cilag International announced the approval of the European Commission for Imbruvica as treatment for the two indications.

Dr. Peter F. Lebowitz, Global Oncology Head at Janssen, said, “By understanding the mechanism of disease and how WM was similar to other B-cell malignancies, our collaboration partner Pharmacyclics was able to pursue this submission for WM, which has the potential to make a very meaningful difference to a group of patients who do not have a sufficient number of treatment options available to them today.” Janssen reported that ibrutinib received Breakthrough Therapy Designation in February last year for patients with WM.

Waldenstrom's macroglobulinemia is a rare, incurable type of B-cell lymphoma that begins with a malignant change to normal B cells. The disease causes the production of excessive amounts of the antibody immunoglobulin M (IgM) which leads to thickening of the blood. The disease leads to excess bleeding, vision difficulties, and nervous system problems. WM affects an estimated 1,000 to 1,500 new patients every year in the U.S., typically between 60 to 70 years of age.

Carl Harrington, President of the International Waldenstrom's Macroglobulinemia Foundation, said, “Waldenstrom's macroglobulinemia is considered an orphan disease. Currently, there are no approved treatment options specifically for WM. The potential approval of a WM-specific treatment will make an immense difference in our patients' lives, offering an FDA-approved option where we previously had none.”

Janssen and Pharmacyclics are currently conducting a clinical development program for Imbruvica, which will include Phase 3 studies in several patient populations.