The Food and Drug Administration (FDA) has expanded the approval of Rituxan injection (rituximab; Genentech and Biogen) to include the treatment of children ≥2 years of age who have the rare disease granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA), in combination with glucocorticoids.
Rituximab is a CD20-directed cytolytic monoclonal antibody first approved in 1997 to treat Non-Hodgkin’s lymphoma and is now approved for multiple indications in adults, including for the treatment of GPA and MPA.
The supplemental Biologics License Application for use in children ≥2 years of age was granted Priority Review in June 2019, and today’s approval was based on a multicenter, open label, phase 2a trial of 25 patients aged between 6 to 17 years.
Patients received either Rituxan or non-US-licensed rituximab. Results demonstrated 14 patients were in remission at the 6-month mark. After 6 months, patients who had not achieved remission or who had progressive disease could receive additional treatment. After 18 months, all 25 patients were reported to be in remission.
The safety profile was found to be consistent in that of adult GPA and MPA patients. The most common side effects in the pediatric study were infections, infusion-related reactions and nausea. Hypogammaglobulinemia has also been observed.
“Rituxan provides a treatment option that has not existed until now for children who suffer from these 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.
Rituxan contains a Boxed Warning for fatal infusion reactions; potentially fatal severe skin and mouth reactions; hepatitis B virus reactivation and progressive multifocal leukoencephalopathy, a rare, serious brain infection that can result in severe disability or death.
For more information visit FDA.gov.
This article originally appeared on MPR