Tuesday, Jun 11, 2019
South San Francisco, CA -- June 11, 2019 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: 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.
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).3 AAV is a form of vasculitis, or blood vessel inflammation, that primarily affects small blood vessels.3 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.1,4 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.1,4 Cases of pediatric onset GPA and MPA are even more rare and are associated with severe, potentially life-threatening symptoms.5
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 with GPA and MPA.
Pemphigus vulgaris (PV): to treat adults with moderate to severe PV.
It is not known if Rituxan is safe or effective in children.
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 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.
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.
1 Medscape. Granulomatosis with Polyangiitis (Wegener Granulomatosis). Available at: https://emedicine.medscape.com/article/332622-overview#showall [Last accessed: March 25, 2019]
2 Brogan P, et al. Pediatric Open-Label Clinical Study of Rituximab for the Treatment of Granulomatosis with Polyangiitis (GPA) and Microscopic Polyangiitis (MPA) [abstract]. Arthritis Rheumatol. 2018; 70 (suppl 10).
3 Genetic and Rare Diseases Information Center (GARD), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH). ANCA-associated vasculitis. Available at: https://rarediseases.info.nih.gov/diseases/13011/anca-associated-vasculitis [Last accessed: March 25, 2019]
4 Medscape. Microscopic Polyangiitis. Available at: https://emedicine.medscape.com/article/334024-overview#showall [Last accessed: March 25, 2019]
5 Iudici M, et al. Childhood-onset granulomatosis with polyangiitis and microscopic polyangiitis: systematic review and meta-analysis. Orphanet J Rare Dis. 2016; 11: 141.