FDA Approves New Drug for Rare Blood Cancer

The Food and Drug Administration (FDA) has approved a new drug to treat a rare and aggressive blood cancer known as mantle cell lymphoma (MCL).

Called Imbruvica, the drug works by inhibiting the enzyme the cancer needs to multiply and spread and is designed to be used in patients who have already received one prior therapy

It is the second drug designated as a breakthrough therapy to receive approval since the passing of the Food and Drug Administration Safety and Innovation Act in 2012 allowed the agency to do so. By placing it in this category, the FDA has indicated that clinical evidence suggests it may represent a significant improvement over current therapies.

"Imbruvica's approval demonstrates the FDA's commitment to making treatments available to patients with rare diseases," Dr. Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, said in a statement. "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."

In a study, 111 participants received Imbruvica daily until their disease progressed or side effects became intolerable. Sixty-six percent saw their cancer either shrink or disappear after treatment, although health officials note that an improvement in survival or disease-related symptoms have not been established.

MCL is a rare form of non-Hodgkin lymphoma, accounting for roughly 6 percent of all non-Hodgkin lymphoma cases in the United States. In most instances, it has already spread to the lymph nodes, bone marrow and other organs by the time it is diagnosed.

The most common side effects included low levels of platelets in the blood, diarrhea, a decrease in infection-fighting white blood cells, anemia, fatigue, musculoskeletal pain, swelling, nausea, bruising, shortness of breath, constipation, rash, abdominal pain, vomiting, upper respiratory infection, kidney problems and the development of other types of cancer.