FDA approves first treatment for children with rare blood vessel diseases


The U.S. Food and Drug Administration (FDA) approved the first treatment for children suffering from rare vasculitis or inflammation of the blood vessels.

According to the FDA, Genentech received the approval for Rituxan (rituximab) injection in combination with glucocorticoids (steroid hormones) to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children 2 years of age and older.

Granulomatosis with polyangiitis (GPA) formerly known as Wegener’s granulomatosis is a rare disease that causes inflammation of the blood vessels in the nose, sinuses, throat, lungs and kidneys.

Microscopic polyangiitis (MPA) is also an uncommon disorder that causes inflammation of the blood vessels in the kidneys, lungs, nerves, skin and joints.

These two types of vasculitis are associated with severe and potentially life-threatening symptoms in children.

Rituxan will achieve an unmet need to treat rare and serious diseases in children

 “The Rituxan application for pediatric GPA and MPA was approved under a priority review and with orphan designation,” said Nikolay Nikolov, M.D., the Associate Director for Rheumatology, Division of Pulmonary, Allergy and Rheumatology Products at the FDA’s Center for Drug Evaluation and Research.

He added that Rituxan will “fulfill an unmet medical need for these rare and serious diseases” and “provides a treatment option that has not existed until now for children” suffering from GPA and MPA.

In a statement, Sandra Horning, M.D., chief medical officer and head of Global Product Development at Genentech said, “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.”

“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,” she added.

The FDA based its approval on data from a clinical trial involving 25 patients (between 6 and 17 years of age) with GPA or MPA. The patients were treated with Rituxan injection combined with a tapering course of oral glucocorticoids. After 18 months, all of the patients were in remission.

The FDA noted that “additional pharmacokinetic (exposure) and safety information supported the use of Rituxan in patients 2 years to 5 years of age with GPA/MPA.”

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