06/12/2019 | Press release | Distributed by Public on 06/12/2019 02:52
Genentech, a member of the Roche Group (OTC: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental Biologics License Application (sBLA) and granted Priority Review for the use of Rituxan® (rituximab), in combination with glucocorticoids (GCC), for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children two years of age and older. GPA and MPA are rare, potentially life-threatening diseases affecting small and medium sized blood vessels.1
'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, M.D., 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 two serious and potentially life-threatening diseases.'
Priority Review Designation is granted to medicines that, if approved, the FDA has determined to have the potential to provide significant improvements in the safety or effectiveness of the treatment of a serious disease.
The sBLA was submitted based on data from the PePRS study, a Phase IIa, global, open-label, single-arm study investigating the safety, pharmacodynamics/pharmacokinetics and exploratory efficacy of intravenous Rituxan in 25 patients with severe GPA or MPA between six and 17 years of age.2 Treatment with four weekly infusions of Rituxan in combination with a tapering course of oral glucocorticoids was assessed in newly diagnosed or relapsing active GPA or MPA pediatric patients.
Rituxan is currently indicated for the treatment of four autoimmune indications. The FDA approved Rituxan for rheumatoid arthritis (RA) in 2006, for the treatment of adults with GPA and MPA in 2011, and for adults with pemphigus vulgaris in 2018. Since 2006, more than 900,000 patients have been treated with Rituxan for autoimmune conditions worldwide. If approved, this would be the first pediatric indication for Rituxan.