USFDA approval of Rituxan, in combination with glucocorticoids, for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in paediatric patients 2 years of age and older

Roche announced that the US Food and Drug Administration (FDA) has approved Rituxan (rituximab), in combination with glucocorticoids, for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in paediatric patients 2 years of age and older. GPA and MPA are rare, potentially life-threatening diseases affecting small and medium sized blood vessels.

The approval is based on data from the PePRS study, a phase IIa, global, open-label, multicentre single-arm study investigating the safety, pharmacokinetics, exploratory efficacy and pharmacodynamic outcomes of intravenous MabThera/Rituxan in 25 patients with active GPA or MPA between 6 and 17 years of age. Treatment with four weekly infusions of MabThera/Rituxan in combination with a tapering course of oral glucocorticoids was assessed in newly diagnosed or relapsing active GPA or MPA paediatric patients. Of the 25 patients in the study, 19 had GPA and 6 had MPA at baseline. Efficacy was an exploratory endpoint and primarily assessed using the Paediatric Vasculitis Activity Score (PVAS). Efficacy assessment showed that 56% of patients achieved PVAS remission by month 6, 92% by month 12, and 100% of patients achieved remission by month 18. The safety profile of MabThera/Rituxan in patients with paediatric GPA and MPA was consistent in type, nature and severity with the known safety profile of MabThera/Rituxan in adult patients with GPA and MPA, rheumatoid arthritis and pemphigus vulgaris.

The FDA previously granted Priority Review to Rituxan for the treatment of GPA and MPA in paediatric patients. In 2011, Rituxan became the first and only therapy approved by the FDA for the treatment of adults with these two rare forms of vasculitis. MabThera/Rituxan is currently indicated for the treatment of four autoimmune conditions and since 2006 more than 900,000 people have been treated with MabThera/Rituxan for autoimmune conditions worldwide. MabThera/Rituxan is not indicated in children less than 2 years of age with GPA or MPA, or in children with conditions outside of GPA and MPA.

Granulomatosis with polyangiitis (GPA) (formerly known as Wegener’s Granulomatosis) and microscopic polyangiitis (MPA) are two types of ANCA-associated vasculitis (AAV). AAV is a form of vasculitis, or blood vessel inflammation, that primarily affects small blood vessels. In general, GPA and MPA both affect the small blood vessels of the kidneys, lungs, sinuses, and a variety of other organs, but the diseases may affect each person differently. Both GPA and MPA are considered rare diseases, with an estimated prevalence globally of 23 to 160 cases per million in the population. Cases of paediatric onset GPA and MPA are even more rare and are associated with severe, potentially life-threatening symptoms.

MabThera (Rituxan in the US) is indicated for the treatment of four autoimmune conditions. MabThera/Rituxan in combination with glucocorticoids, is indicated for the treatment of granulomatosis with polyangiitis (Wegener’s granulomatosis, GPA) and microscopic polyangiitis (MPA) in adults. Rituxan, in combination with glucocorticoids, is also indicated for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in paediatric patients 2 years of age and older.

MabThera/Rituxan in combination with methotrexate, is indicated for the treatment of adults with severe active rheumatoid arthritis (RA) who have had an inadequate response or intolerance to other disease-modifying anti-rheumatic drugs (DMARD), including one or more tumour necrosis factor (TNF) inhibitor therapies. MabThera/Rituxan is indicated for the treatment of adults with moderate to severe pemphigus vulgaris (PV). People with serious infections should not receive MabThera/Rituxan.

MabThera/Rituxan is not indicated in children less than 2 years of age with GPA or MPA, or in children with conditions outside of GPA and MPA.