FDA approves Janssen’s rare blood cancer drug

The US Food and Drug Administration has approved Janssen and Pharmacyclic's rare disease drug, Imbruvica (ibrutinib) to treat patients with a type of aggressive blood cancer called mantle cell lymphoma (MCL). It is the third drug approved to treat MCL and the second drug with breakthrough therapy designation to receive FDA approval.

MCL is a rare form of non-Hodgkin lymphoma and represents about 6% of all non-Hodgkin lymphoma cases in the United States. By the time MCL is diagnosed, it usually has already spread to the lymph nodes, bone marrow and other organs.

"Imbruvica's approval demonstrates the FDA's commitment to making treatments available to patients with rare diseases. The agency worked cooperatively with the companies to expedite the drug's development, review and approval, reflecting the promise of the Breakthrough Therapy Designation program."

Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.

Imbruvica has also been approved under the agency's accelerated approval program, which allows the FDA to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. Plus, Imbruvica has also been given orphan drug status.

The drug's accelerated approval for MCL is based on a clinical trial of 111 patients, who were given Imbruvica daily until their disease progressed or side effects became intolerable. Results showed nearly 66% of participants had their cancer shrink or disappear after treatment (overall response rate).