Posted by Michael Wonder on 27 Sep 2019
FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels
27 September 2019 - The U.S. FDA 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.
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 safety profile in paediatric patients with GPA, formerly known as Wegener's granulomatosis, and MPA was consistent in type, nature and severity with the known safety profile of Rituxan in adult patients with autoimmune diseases, including GPA and MPA. The paediatric 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.
