FDA approves ibrutinib for treatment of mantle cell lymphoma

Today's U.S. Food and Drug Administration (FDA) approval of ibrutinib to treat patients with mantle cell lymphoma (MCL) is a significant advance for patients with this blood cancer.  It was approved as a single agent for treatment of patients with MCL who have received at least one prior therapy. 

Ibrutinib is the second drug with "breakthrough therapy designation" to receive FDA approval. Ibrutinib is a therapy that  targets an enzyme, Bruton's tyrosine kinase (BTK), which promotes growth of  B-cell cancers, including MCL, chronic lymphocytic leukemia (CLL), Waldenstrom's macroglobulinemia (WM), follicular lymphoma, diffuse large B-cell lymphoma, hairy cell leukemia (HCL) and multiple myeloma.

"After the FDA earlier this year designated ibrutinib as a breakthrough therapy for patients with certain forms of blood cancers, we were all hopeful that it was only a matter of time before the therapy was approved to treat these patients, who have few good treatment options," said John Walter, president and CEO of The Leukemia & Lymphoma Society (LLS).

LLS is eager to learn the outcome of FDA's ongoing review of ibrutinib for patients with chronic lymphocytic leukemia (CLL) and Waldenstrom's macroglobulinemia (WM).

"Many patients with MCL and other B-cell cancers do not respond well to standard therapies and new treatments such as ibrutinib are urgently needed," said Louis J. DeGennaro, Ph.D., LLS chief mission officer.  "LLS exists to find cures and ensure access to treatments for blood cancer patients, and any new advance that brings the potential to help save more lives is good news."

LLS funding has supported clinical trials and laboratory studies of ibrutinib for patients with CLL, and more recently for patients with HCL. John C. Byrd, M.D., of the Ohio State University, is a world-renowned CLL expert who leads an LLS-funded research team that has been instrumental in advancing ibrutinib. Other LLS-funded researchers, including Jonathan Friedberg, M.D., of University of Rochester and Jennifer Brown, M.D., Ph.D., of Dana-Farber Cancer Institute, have also played important roles in clinical trials for ibrutinib.

LLS also helped advance another BTK inhibitor, CC-292 (formerly AVL-292), which showed encouraging activity in an early trial for CLL and lymphoma patients, and is now being tested in combination therapies. LLS partnered with Avila Therapeutics through LLS's Therapy Acceleration Program, from March 2010 until Celgene Corporation acquired Avila in January 2012, bringing critical capital and commercialization expertise to the development of this promising agent.