HORSHAM, Penn.—Janssen Biotech, Inc. today announced the U.S. Food and Drug Administration (FDA) has approved IMBRUVICA™ (ibrutinib) capsules for the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. This indication is based on overall response rate (ORR). An improvement in survival or disease-related symptoms has not been established.
IMBRUVICA is one of the first medications to receive FDA approval via the Breakthrough Therapy Designation pathway. Its approval comes just more than four months after the New Drug Application (NDA) submission was completed in late June 2013. IMBRUVICA is being jointly developed and commercialized by Janssen, a Johnson & Johnson company, and Pharmacyclics, Inc.
“Mantle cell lymphoma is a rare, aggressive type of B-cell lymphoma,” said Michael Wang, M.D., Department of Lymphoma/Myeloma, The University of Texas MD Anderson Cancer Center and lead investigator for the pivotal registration trial PCYC-1104. “With IMBRUVICA, we now have a once-daily oral therapy that has been shown to affect the disease. I’m proud to have been involved in this study.”
MCL is an orphan disease. Orphan diseases are characterized by high unmet need and small patient populations affecting fewer than 200,000 people. In the U.S., approximately 2,900 new cases of MCL are diagnosed each year with a median age at diagnosis of 65. MCL typically involves the lymph nodes, but can spread to other tissues, such as the bone marrow, liver, spleen and gastrointestinal tract. This challenging disease is associated with poor prognoses.
“The approval of IMBRUVICA is great news for MCL patients who have received prior therapy and the physicians who treat them,” said William Hait, M.D., Ph.D., global head, research and development, Janssen Research & Development, LLC. “The Breakthrough Therapy Designation helped turbo-charge our timelines – it’s a remarkable process. It’s an excellent example of collaboration between the FDA, Janssen and Pharmacyclics.”
“Breakthrough Therapy Designation is intended to speed up the development and review of treatments to help address serious or life-threatening diseases. It is gratifying to see this early example of the new Breakthrough Therapy Designation pathway meeting its intention – getting promising treatments to patients who are waiting for new options,” said Dr. Ellen Sigal, chair and founder of Friends of Cancer Research, a think tank and advocacy organization based in Washington, DC.
IMBRUVICA was granted three Breakthrough Therapy Designations by the FDA, including relapsed or refractory MCL. IMBRUVICA was approved under the FDA’s Subpart H regulation. Janssen and Pharmacyclics are continuing an extensive clinical development program for IMBRUVICA, including Phase 3 study commitments in this patient population. Additionally, IMBRUVICA has been submitted to the FDA for the treatment of previously treated patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL).
IMBRUVICA works by blocking a specific protein called Bruton’s tyrosine kinase (BTK). Non-clinical studies have shown that blocking BTK inhibits malignant B-cell survival.
The safety and efficacy of IMBRUVICA in patients with MCL who have received at least one prior therapy were evaluated in an open-label, multi-center, single-arm Phase 2 study of 111 treated patients. The primary endpoint was investigator-assessed overall response rate (ORR). Based on investigator assessment, the ORR was 65.8 percent and the median duration of response was 17.5 months. This endpoint was based on responses assessed according to the revised International Working Group (IWG) for non-Hodgkin’s lymphoma (NHL) criteria.
The IMBRUVICA MCL study was published online in The New England Journal of Medicine in June 2013.
Source: Janssen Biotech