FDA Approves Genentech’s Rituxan in Kids with Two Rare Blood Vessel Disorders

September 30, 2019

The U.S. Food and Drug Administration has approved Genentech’s Rituxan in combination with glucocorticoids for the treatment of granulomatosis with polyangiitis and microscopic polyangiitis 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.

Granulomatosis with polyangiitis (GPA), formerly known as Wegener’s granulomatosis, and microscopic polyangiitis (MPA) are two types of a particular 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.

“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, chief medical officer and head of Global Product Development.

The FDA had 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.

Photo: Sandra Horning, chief medical officer and head of Global Product Development.

Author: Rare Daily Staff

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