First treatment for children with rare vasculitis diseases approved

The Rituxan (rituximab) injection to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children two years of age and older in combination with glucocorticoids (steroid hormones) has been approved by the US Food and Drug Administration (FDA).

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, causing serious problems and damage to organs.

“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,” said Nikolay Nikolov, MD, Associate Director for Rheumatology of the Division of Pulmonary, Allergy and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research.

The safety profile in paediatric patients with GPA and MPA was consistent in type, nature and severity with the known safety profile of Rituxan in adult patients with autoimmune diseases. The paediatric clinical trial consisted of 25 patients ages six to 17 years with active GPA and MPA who were treated with Rituxan or non-US-licensed rituximab in an international multicenter, open-label, single-arm, uncontrolled study.

During the clinical trial, after a six-month remission induction phase where patients were treated only with Rituxan or non-US-licensed rituximab and glucocorticoids, patients who had not achieved remission – or who had progressive disease or an uncontrolled flare-up (when disease symptoms suddenly worsen) – could receive additional treatment, including other therapies, at the discretion of the investigator.

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

Additional pharmacokinetic (exposure) and safety information supported the use of Rituxan in patients two years to five years of age with GPA/MPA. 

The FDA granted the approval of Rituxan to Genentech.