SUNNYVALE, Calif., Feb. 12, 2013 /PRNewswire/ -- Pharmacyclics, Inc. (the "Company") (Nasdaq: PCYC) announced today that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to the investigational oral agent ibrutinib monotherapy for the treatment of patients with relapsed or refractory mantle cell lymphoma (MCL) and to ibrutinib monotherapy for the treatment of patients with Waldenstrom's macroglobulinemia (WM), both of which are B-cell malignancies. The Breakthrough Therapy Designation is intended to expedite the development and review of a potential new drug for serious or life-threatening diseases where "preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development."[i] The designation of a drug as a Breakthrough Therapy was enacted as part of the 2012 Food and Drug Administration Safety and Innovation Act. Pharmacylics, together with Janssen, is working with the FDA to determine the implications of this Breakthrough Therapy Designation to the ongoing and planned development and the filing requirements for the use of ibrutinib in patients with MCL and WM. The company expects to finalize the MCL filing prior to year end and will provide guidance on the WM filing after further discussions with the FDA. Pharmacyclics will provide regulatory updates as further information on implementing the requirements with respect to Breakthrough Therapies are developed by the Secretary of Health and Human Services. "This is a historic moment in oncology. We are truly honored to have received this Breakthrough Designation and are pleased for patients and clinicians with the FDA's decision to expedite the development of ibrutinib," said Bob Duggan, CEO and Chairman of the Board. "This compound entered the clinic in 2009 and has demonstrated tremendous clinical progress over the past four years. I would like to thank our collaboration partner, Janssen, for their support. Together we are committed to bringing this new therapeutic to patients and health care providers." Dr. Ellen Sigal, Chair & Founder of Friends of Cancer Research, a think tank and advocacy organization based in Washington, D.C., said, "As an oncology product, ibrutinib receiving the Breakthrough Therapy Designation is an example of progress and hope for patients fighting a range of cancers. This designation shows that the FDA is dedicated to using an 'all hands on deck approach' to work on products that show promise in treating serious and life-threatening diseases. The breakthrough pathway that our organization worked to create is intended to speed up the development and review of treatments that may demonstrate substantial improvement over existing therapies, and ibrutinib is a great example of using this new designation to potentially accelerate patient access to promising treatments." The FDA Breakthrough Therapy Designation for ibrutinib in MCL patients was based on data from clinical and pre-clinical studies. Ibrutinib has the potential to improve the outcome in this serious and life-threatening disease which has a poor prognosis. The company and Janssen have designed a comprehensive development program of ibrutinib in MCL which includes the following studies:- A Phase III randomized, multi-center registration trial of ibrutinib as a monotherapy versus temsirolimus in relapsed or refractory MCL patients who received at least one prior rituximab-containing chemotherapy regimen, RAY (MCL3001). - A Phase III randomized, double-blind, placebo-controlled study of ibrutinib plus bendamustine and rituximab versus placebo plus bendamustine and rituximab in subjects with newly diagnosed MCL, SHINE (MCL3002). - A Phase II single-arm study of ibrutinib as a monotherapy in patients with MCL who received at least one prior rituximab-containing chemotherapy regimen and who progressed after bortezomib therapy, SPARK (MCL2001). - A Phase II single-arm study of ibrutinib as monotherapy in patients with relapsed or refractory MCL (PCYC-1104).