The FDA Approves First Pediatric Treatment for Rare Forms of Vasculitis

According to a story from, the US Food and Drug Administration (FDA) recently approved the drug rituximab (marketed as Rituxan) as a treatment in child patients aged at least two years for two different forms of vasculitis: microscopic polyangiitis and granulomatosis with polyangiitis. The drug is to be used in combination with steroid hormones. This is the first drug that has been approved for children with these rare inflammatory diseases. Rituximab was approved to treat adult patients with these disorders in 2011.

About Vasculitis Disorders

Vasculitis is a group of inflammatory disorders in which blood vessels are destroyed. Both arteries and veins can be affected. The cause of vasculitis can vary and is not always well understood in some cases.  It can be caused by an infection and some cases appear to have a genetic component. It is regarded as an autoimmune disease, but the exact process that triggers an immune response is unknown. Microscopic polyangiitis and granulomatosis with polyangiitis present very similarly, but the microscopic form typically lacks the upper respiratory inflammation (such as sinusitis) that can appear with granulomatosis with polyangiitis. Symptoms include weight loss, fever, kidney disease, bloody stool, abdominal pain, skin conditions such as purpura, arthritis, vision loss, headache, stroke, high blood pressure, bloody cough, gangrene, and nose bleeds. This disease can cause significant system-wide effects that require prompt treatment. This treatment usually involves immunosuppresive drugs such as steroids in order to bring down inflammation. To learn more about vasculitis, click here.

Treating Child Patients

Rituximab earned Orphan Drug designation for the treatment of these diseases and was approved under Priority Review protocols. A clinical trial of rituximab in pediatric patients with granulomatosis with polyangiitis or microscopic polyangiitis showed a similar safety and therapeutic profile to adult patients who were treated in the same manner. 

With this historic approval, children in the US that are facing the life threatening effects of these rare forms of inflammatory vasculitis will now have access to a critical treatment that has the potential to induce disease remission and improve their overall quality of life.

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