14 June 2019 - - The US Food and Drug Administration has accepted US-based Genentech's supplemental Biologics License Application and granted Priority Review for the use of Rituxan (rituximab), in combination with glucocorticoids, for the treatment of granulomatosis with polyangiitis and microscopic polyangiitis in children two years of age and older, the company said.
Genentech, a member of Switzerland's Roche Group (SIX: RO) (OTCQX: RHHBY)
GPA and MPA are rare, potentially life-threatening diseases affecting small and medium sized blood vessels.
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.
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 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.
Granulomatosis with Polyangiitis (formerly known as Wegener's Granulomatosis) and Microscopic Polyangiitis are two types of ANCA-associated vasculitis.
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 in the United States of up to three cases per 100,000 people.
Cases of pediatric onset GPA and MPA are even more rare and are associated with severe, potentially life-threatening symptoms.
Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercialises medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California.