The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Rituxan (rituximab; Genentech) for use in combination with glucocorticoids for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children ≥2 years old.
The sBLA includes data from the phase 2a PePRS study which investigated the safety, pharmacokinetics and efficacy of rituximab in 25 pediatric patients (6-17 years old) with newly diagnosed or relapsing active GPA or MPA. Results of the study showed that 56% and 100% of treated patients achieved remission (defined as a pediatric vasculitis activity score [PVAS] of 0 and oral prednisone or prednisolone equivalent dose of ≤0.2mg/kg/day [max 10mg/day]) by 6 and 18 months, respectively. Overall, the safety profile of rituximab in pediatric patients was found to be similar to that seen in adult GPA/MPA patients, with no new safety signals observed.
“We are committed to delivering new treatment options for rare diseases, such as pediatric GPA and MPA, for which there are currently no approved medicines,” said Sandra Horning, MD, chief medical officer and head of Global Product Development. “We will continue to work closely with the FDA to bring Rituxan to children with these 2 serious and potentially life-threatening diseases.”
Rituxan, CD20-directed cytolytic monoclonal antibody, is already FDA-approved for the treatment of adult patients with GPA/MPA, in combination with glucocorticoids.
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