FDA Expands Approval of Imbruvica for Rare Blood Cancer

gYik8JVToday, the US Food and Drug Administration (FDA) announced that it has expanded approval of Johnson & Johnson and Pharmacyclics’ Imbruvica (ibrutinib) for a rare form of blood cancer.

The agency approved Imbruvica for patients with Waldenstrom’s macroglobulinemia (WM), a rare form of cancer that begins in the body’s immune system. WM is a type of non-Hodgkin lymphoma (NHL), which usually gets worse slowly over time and causes abnormal blood cells, called B lymphocytes (B-cells), to grow within the bone marrow, lymph nodes, liver and spleen. Additionally, in WM abnormal B-cells overproduce a protein known as immunoglobulin M or IgM (macroglobulin) that can lead to excess bleeding, problems with vision and with the nervous system.

Imbruvica was granted Breakthrough Therapy Designation for WM from US health regulators. In addition to WM, Imbruvica is approved for three other indications in the US, including patients with mantle cell lymphoma (MCL) who have received at least one prior therapy, chronic lymphocytic leukemia (CLL) patients who have received at least one prior therapy and CLL patients with 17p deletion.

The FDA’s decision to approve Imbruvica for treatment of WM is based on results from a 63-patient study, which showed that 62 percent of participants had their cancer shrink after treatment, with the duration of response ranging from 2.8 months to approximately 18.8 months.

“Since the first description of Waldenstrom’s macroglobulinemia more than 70 years ago, there has been no approved treatment for this cancer. Rather, doctors relied on therapies borrowed from similar cancers to treat these patients. I am truly grateful to the FDA for their work and dedication of scientists and clinicians at various leading medical centers who diligently worked on the clinical trial that supports Imbruvica as a safe and effective therapy for patients with Waldenstrom’s macroglobulinemia,” said Steven P. Treon, MD, PhD, Director of the Bing Center for Waldenstrom’s Macroglobulinemia at the Dana-Farber Cancer Institute and Associate Professor at Harvard Medical School, and, who led the trial.

In addition to Breakthrough Therapy, the FDA granted Imbruvica priority review and orphan drug designation for treatment of WM. The agency’s approval comes more than two months ahead of its prescription drug user fee goal date.

“Waldenstrom’s macroglobulinemia patients and physicians have been waiting for a treatment specifically studied and approved to treat this rare disease,” said Carol Harrington, President of the International Waldenstrom’s Macroglobulinemia Foundation. “The approval of Imbruvica is an important milestone for the entire global WM community and has the potential to positively impact our patients, their physicians and caregivers.”

Source: US Food and Drug Administration; Janssen Biotech, Inc.

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