Rx for Children with Rare Disorder Approved

FDA approves first treatment for children with rare disease of the blood vessels

(RxWiki News) The US Food and Drug Administration (FDA) has approved the first treatment for children with a rare disorder.

This medication is found under the names Rituxan (brand name) and rituximab (active ingredient). The FDA approved it for use in children (2 years and older) who have rare diseases that lead to inflammation of small blood vessels. The drug was approved to be given in combination with steroid hormones.

Some of the conditions rituximab has been approved to treat include granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). These conditions, marked by the inflammation of small blood vessels, reduce the amount of blood that can flow through the blood vessels.

With limited blood flow, the organs do not get the blood they need.

Rituxan is available as an injection to be administered into the vein by a health care professional. Common side effects may include infections, infusion-related reactions and changes in blood cell counts.

Serious side effects may include infusion-related reactions, severe skin and mouth reactions, hepatitis B virus reactivation and a rare, serious brain infection. This medication includes a boxed warning — the most serious type of warning — about these potentially deadly side effects.

Before this recent approval for use in children, rituximab was approved to treat GPA and MPA in adults back in 2011. Rituxan is also approved to treat a variety of other conditions, including non-Hodgkin's lymphoma.

This medication's approval was granted to Genentech.

Written by Anyssa Garza, PharmD, BCMAS