FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels

FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels

The U.S. Food and Drug Administration today approved Rituxan (rituximab) injection to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children 2 years of age and older in combination with glucocorticoids (steroid hormones). It is the first approved treatment for children with these rare vasculitis diseases, in which a patient’s small blood vessels become inflamed, reducing the amount of blood that can flow through them. This can cause serious problems and damage to organs, most notably the lungs and the kidneys. It also can impact the sinuses and skin.

The most common side effects of Rituxan are infections, infusion-related reactions, abnormally low level of lymphocytes in the blood (lymphopenia) and anemia. Health care professionals are advised to monitor patients for tumor lysis syndrome (a treatment complication where tumor cells are killed off at the same time and released into the bloodstream), cardiac adverse reactions, damage to kidneys (renal toxicity), and bowel obstruction and perforation (small hole formation).

The doctor and patient information for Rituxan contains a boxed warning to draw attention to increased risks of the following: fatal infusion reactions; potentially fatal severe skin and mouth reactions; hepatitis B virus reactivation that may cause serious liver problems, including liver failure and death; and progressive multifocal leukoencephalopathy, a rare, serious brain infection that can result in severe disability or death. This product must be dispensed with a patient Medication Guide that provides important information about the drug’s uses and risks.

For more information, please visit: Rituxan