The FDA has given the green light to rituximab (Rituxan) injection for the treatment of granulomatosis with polyangiitus (GPA) and microscopic polyangiitis (MPA) in children age 2 and older in combination with glucocorticoids. The application was approved under priority review and with orphan designation.
The FDA has given the green light to rituximab (Rituxan, Genentech) injection for the treatment of granulomatosis with polyangiitus (GPA) and microscopic polyangiitis (MPA) in children age 2 years and older in combination with glucocorticoids. The application was approved under priority review and with orphan designation.
These rare vasculitis diseases cause a patient's small blood vessels to become inflamed, reducing the amount of blood that can flow through them. These conditions can also cause damage to organs, especially the lungs and kidneys. This FDA announcement marks the first approved treatment for children with the diseases.
The pediatric clinical trial included 25 patients from ages 6 to 17 years with active GPA and MPA, all of whom were given methylprednisolone prior to starting treatment. At the 6-month mark, 15 of the patients were in remission. At this point, patients who had not achieved remission, had progressive disease, or had an uncontrolled flare-up could receive additional treatment including other therapies at the discretion of the investigator.
After 18 months, all 25 patients achieved remission. The most common adverse effects were infections, infusion-related reactions, and nausea. Hypogammaglobulinemia (reduced serum immunoglobulin levels) has also been observed in pediatric GPA and MPA patients treated with the study drugs.
The safety profile in pediatric patients with the disease was consistent in type, nature, and severity with the known safety profile of Rituxan in adult patients with autoimmune diseases. The doctor and patient information contains a boxed warning for increased risks of fatal infusion reactions, potentially fatal skin and mouth reactions, hepatitis B virus reactivation, and progressive multifocal leukoencephalopathy.
Rituxan was first approved for adult patients with GPA and MPA in 2011. It is also approved to treat 4 other diseases, including Non-Hodgkin's lymphoma.
FDA approves first treatment for children with rare diseases that cause inflammation of small blood vessels [news release]. Silver Spring, MD; September 27, 2019: FDA website. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-children-rare-diseases-cause-inflammation-small-blood-vessels. Accessed September 27, 2019.