Janssen announces submission of IMBRUVICA sNDA to FDA for WM treatment

Janssen Research & Development, LLC ("Janssen") today announced the submission of a supplemental New Drug Application (sNDA) for IMBRUVICA^® (ibrutinib) to the U.S. Food and Drug Administration (FDA) by its strategic partner Pharmacyclics, Inc. If approved, this latest regulatory submission will become the fourth indication for IMBRUVICA, adding the treatment of patients with Waldenstrom's macroglobulinemia (WM). WM is a rare type of B-cell lymphoma for which there are no treatment options specifically approved in the U.S. IMBRUVICA received FDA Breakthrough Therapy Designation in February 2013 for patients with WM and is being jointly developed and commercialized by Janssen Biotech Inc. and Pharmacyclics.

"We are committed to bringing our medicines to new patient populations, large and small, who may benefit from them," said Peter F. Lebowitz, M.D., Ph.D., Global Oncology Head, Janssen. "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."

"Waldenstrom's macroglobulinemia is considered an orphan disease. Currently, there are no approved treatment options specifically for WM," said Carl Harrington, President of the International Waldenstrom's Macroglobulinemia Foundation. "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."

WM (also known as lymphoplasmacytic lymphoma) is a slow-growing, incurable, rare type of B-cell lymphoma for which no established standard of care – or approved therapeutic – exists. In the U.S., there are approximately 1,000 to 1,500 new cases each year and the median age at diagnosis is 60-70 years of age. WM begins with a malignant change to the B cell, a type of white blood cell (lymphocyte), during its maturation so that it continues to reproduce more malignant B cells. WM cells make large amounts of a certain type of antibody (immunoglobulin M, or IgM). Antibodies such as IgM normally help the body to fight infection. Excess IgM causes the blood to thicken and causes many of the symptoms of WM, including excess bleeding, problems with vision and nervous system problems.

The currently approved indications for IMBRUVICA are: 1) for the treatment of patients with chronic lymphocytic leukemia (CLL) who have received at least one prior therapy, 2) for the treatment of CLL patients with del 17p, a genetic mutation that occurs when part of chromosome 17 has been lost, and 3) for the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. Accelerated approval was granted for the MCL indication based on overall response rate (ORR). Improvements in survival or disease-related symptoms have not been established. Continued approval for the MCL indication may be contingent upon verification of clinical benefit in confirmatory trials. IMBRUVICA was granted Breakthrough Therapy Designation by the FDA for the MCL, WM and CLL with del 17p indications.

Janssen and Pharmacyclics are continuing an extensive clinical development program for IMBRUVICA, including Phase 3 study commitments in multiple patient populations.