FDA Approves Genentech's Rituxan to Treat Two Rare Disorders

SILVER SPRING, Md., April 19, 2011 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration today approved Rituxan (rituximab), in combination with glucocorticoids (steroids), to treat patients with Wegener's granulomatosis (WG) and microscopic polyangiitis (MPA), two rare disorders that cause blood vessel inflammation (vasculitis).

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Vasculitis in patients with WG and MPA can lead to tissue damage. WG mostly affects the respiratory tract (sinuses, nose, trachea, and lungs) and kidneys, while MPA commonly affects the kidneys, lungs, nerves, skin, and joints. Both of these diseases affect people of all ages and ethnicities, and both genders. The causes of these disorders are unknown, and both are considered orphan diseases because they each affect less than 200,000 people in the United States.

"This new indication for Rituxan provides the first approved therapy for these two orphan diseases," said Curtis Rosebraugh, M.D., M.P.H., director of the Office of Drug Evaluation II in the FDA's Center for Drug Evaluation and Research.

Rituxan is an antibody that is manufactured through biotechnology methods. The drug works by greatly reducing the number of specific immune cells in the blood, known as B cells.

The safety and effectiveness of Rituxan was demonstrated in a single controlled trial, in which 197 patients with WG or MPA were assigned at random to receive either Rituxan plus glucocorticoids once a week for four weeks or oral cyclophosphamide plus glucocorticoids daily to induce remission. After six months, 64 percent of patients treated with Rituxan had complete remission compared to 53 percent of patients treated with cyclophosphamide.

Retreatment with Rituxan was not formally evaluated; therefore, the safety and efficacy of retreatment with subsequent courses of Rituxan has not been established. More data are needed to determine the safety of more than one course of Rituxan and long term safety of use of Rituxan in patients with WG and MPA. These questions will be further evaluated in a required post-marketing study.

Rituxan carries a Boxed Warning for infusion reactions, which can occur during infusion or within 24 hours afterwards. Other Boxed Warnings for Rituxan include rashes and sores in the skin and mouth (severe mucocutaneous reactions); and progressive multifocal leukoencephalopathy, a brain infection that generally is fatal. Rituxan is not recommended for use in patients with severe, active infections.

The most common side effects in study participants with WG and MPA included infection, nausea, diarrhea, headache, muscle spasms, and anemia.

Rituxan, which has been marketed since 1997, is also indicated for the treatment of patients with non-Hodgkin's lymphoma, chronic lymphocytic leukemia, and rheumatoid arthritis.

Rituxan is manufactured by San Francisco-based Genentech, a member of the Roche Group.

For more information:

Approved Drugs: Questions and Answers
http://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm054420.htm

National Institute of Allergy and Infectious Diseases: Wegener's Granulomatosis
http://www.niaid.nih.gov/topics/wegeners/Pages/Default.aspx

Office of Rare Disease Research: Microscopic Polyangiitis
http://rarediseases.info.nih.gov/GARD/Condition/3652/Microscopic_polyangiitis.aspx

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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SOURCE U.S. Food and Drug Administration