Friday, Sep 27, 2019
Rituxan is the first and only FDA-approved treatment for pediatric patients 2 years of age and older living with granulomatosis with polyangiitis or microscopic polyangiitis
These two forms of vasculitis in children are rare and associated with severe, potentially life-threatening symptoms
This approval is the first pediatric indication for Rituxan
South San Francisco, CA -- September 27, 2019 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. 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 pediatric patients 2 years of age and older. GPA and MPA are rare, potentially life-threatening diseases affecting small- and medium-sized blood vessels.
“Rituxan is now approved as the first and only medicine for pediatric patients living with GPA and MPA, two potentially life-threatening blood vessel disorders which are rare in children,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “Today’s approval is a result of our ongoing commitment to working with the FDA to develop medicines for pediatric patients with rare diseases where there is a serious unmet need.”
The approval is based on data from the PePRS study, a Phase IIa, global, open-label, multi-center, single-arm study investigating the safety, pharmacokinetics, exploratory efficacy and pharmacodynamic outcomes of intravenous Rituxan in 25 patients with active GPA or MPA between 6 and 17 years of age. Treatment with four weekly infusions of Rituxan or non-U.S.-licensed rituximab in combination with a tapering course of oral glucocorticoids was assessed in newly diagnosed or relapsing active GPA or MPA pediatric 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 Pediatric 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 Rituxan in patients with pediatric GPA and MPA was consistent in type, nature and severity with the known safety profile of Rituxan in adult patients with GPA, MPA, rheumatoid arthritis and pemphigus vulgaris.
The FDA previously granted Priority Review to Rituxan for the treatment of GPA and MPA in pediatric 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. Rituxan is currently indicated for the treatment of four autoimmune conditions and since 2006 more than 900,000 people have been treated with Rituxan for autoimmune conditions worldwide. 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.
About Granulomatosis with Polyangiitis and Microscopic Polyangiitis
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 in the United States of up to 3 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.
What autoimmune diseases does Rituxan treat?
Rheumatoid arthritis (RA): with another prescription medicine called methotrexate, to reduce the signs and symptoms of moderate to severe active RA in adults, after treatment with at least one other medicine called a tumor necrosis factor (TNF) antagonist has been used and did not work well enough.
Granulomatosis with Polyangiitis (GPA) (Wegener’s Granulomatosis) and Microscopic Polyangiitis (MPA): with glucocorticoids, to treat adults and pediatric patients 2 years of age and older with GPA and MPA.
Pemphigus vulgaris (PV): to treat adults with moderate to severe PV.
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.
Important Side Effect Information
What is the most important information patients should know about Rituxan?
Rituxan can cause serious side effects that can lead to death, including:
Before receiving Rituxan, patients should tell their healthcare provider if they:
What are the possible side effects of Rituxan?
Rituxan can cause serious and life‐threatening side effects, including:
What are the most common side effects during treatment with Rituxan?
The most common side effects of Rituxan include:
In adult patients with GPA or MPA, the most common side effects of Rituxan also include:
Other side effects include:
These are not all of the possible side effects with Rituxan. For more information, ask a doctor or pharmacist.
Contact a doctor for medical advice about side effects. Report side effects to the FDA at (800) FDA ‐ 1088 or http://www.fda.gov/medwatch. Patients may also report side effects to Genentech at (888) 835 ‐ 2555.
Please see the Rituxan Prescribing Information and Medication Guide including Most Serious Side Effects for additional Important Side Effect Information at http://www.rituxan.com.
Genentech and Biogen collaborate on Rituxan in the United States, and Roche markets MabThera in the rest of the world, except Japan, where Rituxan is co-marketed by Chugai and Zenyaku Kogyo Co. Ltd.
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Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes 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. For additional information about the company, please visit http://www.gene.com.