FDA News

FDA expands Rituxan indication for children with vasculitis diseases

The FDA has approved intravenous Rituxan for the treatment of granulomatosis with polyangiitis and microscopic polyangiitis in children 2 years and older in combination with glucocorticosteroids.

This decision extends the previous indication for Rituxan (rituximab, Genentech) and represents the first approved treatment for children with these rare vasculitis diseases. Rituxan was first approved in 2011 to treat adult patients with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA).

“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 the release.

The FDA based its decision on data from an international multicenter, open-label, single-arm, uncontrolled study examining safety, pharmacokinetics, exploratory efficacy and pharmacodynamic outcomes of intravenous Rituxan in 25 patients aged 6 to 17 years with GPA and MPA. All patients received methylprednisolone prior to commencing treatment.

According to study results, 14 patients achieved remission at 6 months and all 25 patients achieved remission after 18 months.

Sandra Horning, MD
Sandra Horning

“Rituxan is now approved as the first and only medicine for pediatric patients living with GPA and MPA, two potentially life-threatening blood vessel disorders which are rare in children,” Sandra Horning, MD, chief medical officer and head of global product development, said in a press release. “[This] approval is a result of our ongoing commitment to working with the FDA to develop medicines for pediatric patients with rare diseases where there is a serious unmet need.”

The FDA noted that the most common adverse events associated with Rituxan in pediatric patients were infections, infusion-related reactions and nausea; however, hypogammaglobulinemia had also been observed in pediatric patients with GPA and MPA who had been treated with the study products.

Additionally, the FDA cautioned clinicians to monitor patients for tumor lysis syndrome, cardiac adverse reactions, damage to kidneys and bowel obstruction perforation. The medication contains a boxed warning about increased risks of fatal infusion reactions; potentially fatal severe skin and mouth reactions; hepatitis B virus reactivation that may cause serious liver problems, including liver failure and death; and progressive multifocal leukoencephalopathy.

The FDA has approved intravenous Rituxan for the treatment of granulomatosis with polyangiitis and microscopic polyangiitis in children 2 years and older in combination with glucocorticosteroids.

This decision extends the previous indication for Rituxan (rituximab, Genentech) and represents the first approved treatment for children with these rare vasculitis diseases. Rituxan was first approved in 2011 to treat adult patients with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA).

“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 the release.

The FDA based its decision on data from an international multicenter, open-label, single-arm, uncontrolled study examining safety, pharmacokinetics, exploratory efficacy and pharmacodynamic outcomes of intravenous Rituxan in 25 patients aged 6 to 17 years with GPA and MPA. All patients received methylprednisolone prior to commencing treatment.

According to study results, 14 patients achieved remission at 6 months and all 25 patients achieved remission after 18 months.

Sandra Horning, MD
Sandra Horning

“Rituxan is now approved as the first and only medicine for pediatric patients living with GPA and MPA, two potentially life-threatening blood vessel disorders which are rare in children,” Sandra Horning, MD, chief medical officer and head of global product development, said in a press release. “[This] approval is a result of our ongoing commitment to working with the FDA to develop medicines for pediatric patients with rare diseases where there is a serious unmet need.”

The FDA noted that the most common adverse events associated with Rituxan in pediatric patients were infections, infusion-related reactions and nausea; however, hypogammaglobulinemia had also been observed in pediatric patients with GPA and MPA who had been treated with the study products.

Additionally, the FDA cautioned clinicians to monitor patients for tumor lysis syndrome, cardiac adverse reactions, damage to kidneys and bowel obstruction perforation. The medication contains a boxed warning about increased risks of fatal infusion reactions; potentially fatal severe skin and mouth reactions; hepatitis B virus reactivation that may cause serious liver problems, including liver failure and death; and progressive multifocal leukoencephalopathy.