FDA grants approval for Rituxan in treatment of two orphan diseases

Rituxan, manufactured by Genentech, a member of the Roche Group, received U.S. Food and Drug Administration approval, in combination with steroids.

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Kelsey Kaustinen

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Internal Medicine

SILVER SPRING, Md.—Rituxan, manufactured by Genentech, amember of the Roche Group, received U.S. Food and Drug Administration (FDA) approval,in combination with steroids. The drug is meant to treat Wegener'sgranulomatosis (WG) and microscopic polyangiitis (MPA), two orphan diseasesthat cause blood vessel inflammation (vasculitis).

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

Rituxan (rituximab) has been marketed since 1997, and isalso indicated for treating patients with non-Hodgkin's lymphoma, chroniclymphocytic leukemia and rheumatoid arthritis is an antibody, manufacturedthrough biotechnology methods. It works by reducing the quantity of specificimmune cells in the blood, known as B cells.

In patients suffering from WG and MPA, vasculitis can leadto tissue damage. WG involves inflammation of blood vessels, and mostly affectsthe respiratory tract—sinuses, nose, trachea and lungs—as well as the kidneys.Though the cause of the disease is unknown, WG is thought to be an autoimmunedisorder. It is most common in middle-aged adults and rare in children, thoughcases have been seen in infants as young as three months. Symptoms includefrequent sinusitis, night sweats, chronic ear infections, and sore around thenose.

MPA most commonly affects the kidneys, lungs, nervoussystem, skin and joints, though the cause of the disease is not known. The mostcommon clinical manifestations of MPA, according to the Johns HopkinsVasculitis Center, are kidney inflammation, weight loss, skin lesions, nervedamage and fevers. Both WG and MPA affect both men and women of all ages andethnicities, and are considered to be orphan diseases since they each affectfewer than 200,000 people in the U.S.

Rituxan was tested for safety and efficacy in a singlecontrolled trial of 197 patients with WG or MPA. Patients received eitherRituxan and glucocorticoids once a week for four weeks or oral cyclophosphamideplus glucocorticoids daily in order to induce remission. At the end of sixmonths, 64 percent of patients who received Rituxan displayed completeremission, compared to the 53 percent of patients who receivedcyclophosphamide.

The most common side effects seen in study participantsincluded nausea, headache, infection, anemia, diarrhea and muscle spasms.Rituxan has a Boxed Warning for infusion reactions, as well as for rashes andsores in the skin and mouth and progressive multifocal leukoencephalopathy, abrain infection that is usually fatal. The drug is not recommended for patientswith severe and active infections.

Rituxan was approved by the FDA for maintenance therapy inpatients with previously untreated follicular, CD-20 positive, B-cellnon-Hodgkin lymphoma on January 28, 2011, and was approved for the treatment ofchronic lymphocytic leukemia on February 18, 2010.

FDA grants approval for Rituxan in treatment of two orphan diseases

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Kelsey Kaustinen

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