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FDA Approves First Treatment Option for Granulomatosis With Polyangiitis in Children

September 27, 2019

The FDA has approved the first injectable drug for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children 2 years of age and older. Treatment is to be used in combination with glucocorticoids (steroid hormones).  

The approval of the drug, Rituxan (rituximab; Genentech), was based on an international multicenter, open-label, single-arm, uncontrolled study that consisted of 25 patients ages 6-17 years old with active GPA and MPA. The trial participants were treated with Rituxan or non-US-licensed rituximab. After 6 months of the trial, 14 patients achieved remission, and after 18 months, all 25 patients in the trial were in remission.  

The most commonly reported side effects of Rituxan included:

  • infections;
  • infusion-related reactions;
  • abnormally low level of lymphocytes in the blood; and,
  • anemia.

The FDA said that health care professionals should monitor patients for tumor lysis syndrome, cardiac adverse reactions, damage to kidneys, and bowel obstructions and perforation.  

Originally, Rituxan was approved to treat adult patients with GPA and MPA in 2011. Additionally, it is approved to treat four additional diseases.  

“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,” 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, said in a press release. 

Julie Gould  


The US Food and Drug Administration. FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels [press release]. FDA website. September 27, 2019.

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