FDA Approves Option for Granulomatosis With Polyangiitis

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

The drug is the first treatment for children with these diseases to be approved by the FDA and was first approved for the treatment of adults with GPA and MPA in 2011.

The safety of Rituxan was assessed in an international multicenter, open-label, single-arm, uncontrolled study involving 25 patients aged 6 to 17 years with active GPA and MPA treated with Rituxan or with non-US-licensed rituximab. After 6 months of treatment, 14 of the patients were in remission. At 18 months, all 25 were in remission.

The most common adverse effects observed in the pediatric study were infections, infusion-related reactions, and nausea. Rituxan includes a boxed warning of increased risks of fatal infusion reactions, potentially fatal severe skin and mouth reactions, hepatitis B virus reactivation, and progressive multifocal leukoencephalopathy.

—Michael Potts

Reference:

FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels [press release]. Silver Spring, MD: US Food and Drug Administration; September 27, 2019. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-children-rare-diseases-cause-inflammation-small-blood-vessels.