FDA Approves Rituximab for Pediatric GPA, MPA

The US Food and Drug Administration (FDA) has approved add-on rituximab (Rituxan) injection for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children aged 2 years and older.

The indication, which requires the drug be used in combination with glucocorticoids, is the first approved therapy for children with the rare vasculitis conditions. Characteristics of the disease include inflamed small blood vessels, which reduce the flow of blood and can ultimately damage organs such as the lungs and kidneys.

The benefit of rituximab in both GPA and MPA was evidenced in a pediatric clinical trial involved 25 patients aged 6-17 years old with an active form of the disease. Patients were treated with rituximab or a non-US-licensed form of the therapy in an international, multicenter, open-label, single-arm, uncontrolled assessment.

Investigators observed 14 of the patients reached remission at 6 months of rituximab plus glucocorticoid treatment. At 18 months, all 25 patients had achieved remission. The safety profile of the therapy in pediatric patients was consistent with the known profile of rituximab in adult patients with autoimmune diseases including GPA and MPA.

The Genentech therapy was approved to treat adult patients with GPA and MPA in 2011, and has been approved for the treatment of 9 conditions. It was first introduced to the US market in 1997, when it was approved for patients with non-Hodgkin’s lymphoma.

Rituximab was previously granted priority review and orphan drug designations for these pediatric indications.