Application Filed with U.S. FDA for use of Imbruvica to treat WM...more...
10.21.2014 - 6.30.2015
If approved - this will be the FIRST drug approved specifically for WM!
A historic day for WMers
Today is an important day for WMers. As you’ll read in the announcement below, Pharmacyclics & Janssen have filed an application with the U.S. Food & Drug Administration (FDA) for approval of Imbruvica (ibrutinib) for the treatment of Waldenström’s macroglobulinemia in the U.S. If this application is approved, it will be the very first time a drug has been approved for our orphan disease and a moment WMers have been waiting for since our disease was first diagnosed in 1944. Approval by the FDA will make it easier for WMers to obtain Imbruvica and to receive reimbursement for it.
While recognition by the FDA will be an historic step, we are still far from the goal of finding a cure for our disease. Imbruvica is not a cure. Imbruvica doesn’t work for everyone, and it won’t replace all the other treatments we’ve come to rely upon. But the application to the FDA is tangible proof that we’re making progress in getting time and attention devoted to our orphan disease.
We can’t afford to relax and become complacent. We need to build upon this news and continue to fund the support our members need once they receive a diagnosis of WM as well as our relentless search for a cure.
IMBRUVICA® (ibrutinib) Supplemental New Drug Application Submitted to the U.S. FDA for Waldenström’s macroglobulinemia?Latest submission seeks approval for additional blood cancer indication
RARITAN, NJ, October 20, 2014 – 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 Waldenström’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.”
“Waldenström’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 lymphoma1 for which no established standard of care – or approved therapeutic – exists.2,3 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.1,4 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.5
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.6 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.6 IMBRUVICA was granted Breakthrough Therapy Designation by the FDA for 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.
Please consider a donation to the IWMF today to continue our search for a cure and go to http://www.iwmf.com/donate/index.aspx
Thank you and stay well,
P.S. For our European WMers, on October 17, 2014, Pharmacyclics and Janssen announced the approval of Imbruvica for 28 European countries for CLL (chronic lymphocyte leukemia) and MCL (mantle cell lymphoma). Approval for these diseases occurred in the U.S. on July 28, 2014. See http://ir.pharmacyclics.com/releasedetail.cfm?ReleaseID=876757 for more details on the European approval.