FDA Approves Treatment for Small Children with Rare Diseases Impacting Blood Vessels

Approval comes after wildly successful clinical trial

The U.S. Food and Drug Administration recently 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). 

The approval creates 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 Rituxan application for pediatric GPA and MPA was approved under a priority review, and with orphan designation, to fulfil an unmet medical need for these rare and serious diseases. Rituxan provides a treatment option that has not existed until now for children who suffer from these diseases,” said Nikolay Nikolov, M.D., associate director for rheumatology of the Division of Pulmonary, Allergy and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research.

The pediatric clinical trial consisted of 25 patients ages 6 to 17 years with active GPA and MPA who were treated with Rituxan or non-U.S.-licensed rituximab in an international multicenter, open-label, single-arm, uncontrolled study. All patients were given methylprednisolone prior to starting treatment.

In total, 14 of the patients were in remission at the six-month mark. After 18 months, all 25 patients were in remission. 

SOURCE: FDA

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