Rituximab Gains First Pediatric Approval

September 30, 2019

For granulomatosis with microscopic polyangiitis in children.

The FDA has approved the use of Genentech’s rituximab (Rituxan) intravenous infusion, in combination with glucocorticoids, for the treatment of granulomatosis (GPA) with microscopic polyangiitis (MPA) in pediatric patients two years of age and older. 

Rituxan is a CD20-directedcytolytic antibody also indicated for the treatment of non-hodgkin’s lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis, moderate to severe pemphigus vulgaris, and GPA with MPA in adult patients. 

“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, MD, chief medical officer and head of Global Product Development, in a statement. 

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The approval is based upon results of the Phase IIa, global, open-label, multi-center, single-arm study investigating the product’s safety and efficacy. 25 patients with active GPA or MPA between the ages of six and 17 years of age were treated with four weekly infusions of rituximab or non-U.S.-licensed rituximab in combination with a tapering course of oral glucocorticoids. At the start of the study, 19 participating patients had GPA, while six had MPA. 

Efficacy results were measured as an exploratory endpoint and assessed using the Pediatric Vasculitis Activity Score (PVAS). Fifty-six percent of patients achieved PVAS remissions by month six, 92% by month 12, and 100% by month 18. 

Safety was consistent in type, nature, and severity with the known safety profiles of rituximab in adult patients with GPA, MPA, rheumatoid arthritis, and pemphigus vulgaris.

Rituximab comes with a boxed warning of potential fatal infusion-related reactions that occur within 24 hours of administration, usually in response to the first infusion; severe mucocutaneous reactions, some fatal; Hepatitis B virus reactivation, sometimes resulting in fulminant hepatitis, hepatic failure, and death; and progressive multifocal leukoencephalopathy resulting in death. 

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Other warnings include tumor lysis syndrome, infections, cardiac adverse reactions, renal toxicity, bowel obstruction and perforation, and embryo-fetal toxicity. Live virus immunizations are not recommended prior to or during treatment with rituximab. 

Adverse events reported across all indications include fever, lymphopenia, chills, infection, asthenia, infusion-related reactions, neutropenia, upper respiratory tract infection, nasopharyngitis, urinary tract infection, bronchitis, nausea, diarrhea, headache, muscle spasm, anemia, peripheral edema, and depression.

 

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