New Indication for Roche’s Actemra Means First FDA-Approved Drug to Treat Giant Cell Arteritis

The US Food and Drug Administration (FDA) on Monday expanded the approved use of Roche’s Actemra (tocilizumab) to treat adults with giant cell arteritis, making it the first FDA-approved therapy for this condition.

The efficacy and safety of subcutaneous (injected under the skin) Actemra for giant cell arteritis were established in a double-blind, placebo-controlled study with 251 patients with giant cell arteritis. A greater proportion of patients receiving subcutaneous Actemra with standardized prednisone regimens achieved sustained remission from Week 12 through Week 52 as compared to patients receiving placebo with standardized prednisone regimens.

FDA noted that the overall safety profile observed in the Actemra treatment groups was “generally consistent with the known safety profile of Actemra,” which carries a boxed warning for serious infections. Actemra won a breakthrough therapy designation and priority review for this indication.

"We expedited the development and review of this application because this drug fulfills a critical need for patients with this serious disease who had limited treatment options," said Badrul Chowdhury, MD, PhD, director of the Division of Pulmonary, Allergy, and Rheumatology Products in FDA’s Center for Drug Evaluation and Research.

According to FDA, giant cell arteritis is a form of vasculitis, a group of disorders that results in inflammation of blood vessels, which causes arteries to narrow or become irregular. In giant cell arteritis, the vessels most involved are those of the head, especially the temporal arteries (located on each side of the head). For this reason, the disorder is sometimes called temporal arteritis. Standard treatment involves high doses of corticosteroids that are tapered over time.

In 2010, subcutaneous Actemra was approved by FDA for the treatment of moderate to severely active rheumatoid arthritis. It was also previously approved for the treatment of moderate to severely active rheumatoid arthritis, systemic juvenile idiopathic arthritis and polyarticular juvenile idiopathic arthritis.