GPA and MPA are rare and potentially life-threatening diseases, which affect small and medium-sized blood vessels.
The FDA approval was based on data from the PePRS phase IIa, a global, open-label, multicentre single-arm study that assessed the safety, pharmacokinetics, exploratory efficacy and pharmacodynamic outcomes of intravenous MabThera/Rituxan in 25 patients with active GPA or MPA between six and 17 years of age.
Treatment with four weekly infusions of MabThera/Rituxan in combination with a tapering course of oral glucocorticoids was evaluated in newly diagnosed or relapsing active GPA or MPA paediatric patients.
The 19 had GPA and 6 had MPA at baseline, of the 25 patients evaluated in the study. According to the company, the efficacy assessment demonstrated that 56% of patients achieved PVAS remission by month 6, 92% by month 12, and 100% of patients achieved remission by month 18.
Roche chief medical officer and global product development head Dr Sandra Horning said: “Rituxan is now approved as the first and only medicine for paediatric patients living with GPA and MPA, two potentially life-threatening blood vessel disorders which are rare in children.
“Today’s approval is a result of our ongoing commitment to working with the FDA to develop medicines for paediatric patients with rare diseases where there is a serious unmet need.”
Earlier, the FDA awarded priority review to Rituxan to treat GPA and MPA in paediatric patients. At present, MabThera/Rituxan can be used to treat four autoimmune conditions.
Rituxan became the first and only FDA approved treatment for adults with GPA and MPA forms of vasculitis in 2011.
MabThera/Rituxan in combination with methotrexate can also be used to treat adults with severe active rheumatoid arthritis (RA) who have had an inadequate response or intolerance to other disease-modifying anti-rheumatic drugs (DMARD).
Earlier this month, Roche secured a breakthrough therapy designation (BTD) from the US Food and Drug Administration (FDA) for its Gazyva (obinutuzumab) to treat adults with lupus nephritis.
Gazyva is an engineered monoclonal antibody developed to attach to CD20, a protein present only on certain types of B-cells.