Janssen Research & Development, LLC announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designations for the investigational oral agent ibrutinib as a monotherapy for two B-cell malignancies: in patients with relapsed or refractory mantle cell lymphoma (MCL) who have received prior therapy, and in patients with Waldenstrom's macroglobulinemia (WM).

Enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy Designation is intended to expedite the development and review time for a potential new medicine "to treat a serious or life-threatening disease or condition and 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."

"We are pleased that the FDA has granted two Breakthrough Therapy Designations for ibrutinib as the designation represents a major leap forward in accelerating drug development timelines," said Paul Stoffels , M.D., Chief Scientific Officer and Worldwide Chairman, Pharmaceuticals, Johnson & Johnson.

"We are committed to realising the full potential of ibrutinib for patients with mantle cell lymphoma, Waldenstrom's macroglobulinemia, as well as other B-cell malignancies, and will work with Pharmacyclics and the FDA to ensure the clinical development program for ibrutinib continues to move forward as quickly as possible."

Janssen Biotech, Inc. and Pharmacyclics entered a collaboration and license agreement in December 2011 to co-develop and co-commercialize ibrutinib. The filing for ibrutinib in MCL is expected to be made prior to the end of 2013 and discussions with the FDA about filing in WM continue. The implications of Breakthrough Therapy Designation cannot be determined at this time. Janssen and Pharmacyclics are working with the FDA to determine any potential implications of the Breakthrough Therapy Designations to the ongoing and planned development activities.

"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," said Dr. Ellen Sigal , Chair and Founder of Friends of Cancer Research, a think tank and advocacy organization based in Washington DC.

"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."

Source: Janssen