The U.S. Food and Drug Administration has granted the new Breakthrough Therapy designation to ibrutinib monotherapy (an oral agent) for the treatment of patients with relapsed or refractory mantle cell lymphoma (MCL) and for the treatment of patients with Waldenstrom's macroglobulinemia (WM).
The designation, enacted as part of the FDA's 2012 Safety and Innovation Act, was created to expedite the development and review time of a potential new drug for serious or life-threatening disease where early clinical evidence suggests the drug may demonstrate substantial improvement compared with existing therapies.
“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,” Ellen Sigal, PhD, Chair and Founder of the Washington, D.C.-based think tank Friends of Cancer Research, said in a news release.
This designation for ibrutinib for MCL treatment was based on data from clinical and preclinical studies. The drug is being co-developed and co-commercialized by Janssen Biotech, Inc. and Pharmacyclics. And, Janssen is planning the following studies for the drug:
* RAY (MCL3001): A Phase III randomized, multicenter registration trial of ibrutinib as a monotherapy versus temsirolimus in relapsed or refractory MCL patients who have received at least one prior rituximab-containing chemotherapy regimen.
* SHINE (MCL3002): 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.
* SPARK (MCL2001): 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.
* PCYC-1104: A Phase II single-arm study of ibrutinib as monotherapy in patients with relapsed or refractory MCL.
Filing for the drug for MCL treatment is expected to be made by the end of the year, and a timeline has not yet been announced for filing for the drug for WM treatment.