FDA approves first drug to treat giant cell arteritis

Adults with giant cell arteritis – a form of vasculitis, or inflammation of blood vessels – received good news this week with the US FDA. Extend the approved use of subcutaneous Actemra (tocilizumab) to treat adults suffering from the condition. This new indication provides the first therapy approved by the FDA, specific for this type of vasculitis.

“We have streamlined the development and review of this application because this drug meets a critical need for patients with this serious illness who had limited treatment options,” said Badrul Chowdhury, MD, Ph.D., director of the Pulmonary Division, And rheumatology products at the FDA’s center for drug evaluation and research.

Giant cell arteritis is a form of vasculitis that causes inflammation of the blood vessels. This inflammation causes the arteries to narrow or become irregular, hindering proper blood flow. In giant cell arteritis, the vessels most involved are those of the head, especially the temporal arteries (located on each side of the head). The standard treatment of the disorder is typically high doses of corticosteroids that tighten over time.

Actemra’s approval for giant cell arteritis was based on the results of a double-blind, placebo-controlled study of 251 patients with giant cell arteritis. The primary endpoint of efficacy was the proportion of patients achieving sustained remission from week 12 to week 52. Sustained remission was defined as absence of giant cell arteritis symptoms, normalization of laboratory inflammatory tests, and decrease of the use of prednisone, a higher proportion of patients receiving subcutaneous Actemra with normalized prednisone regimens achieved sustained remission from week 12 to week 52 compared to patients receiving placebo with normalized prednisone regimens. The cumulative dose of prednisone was lower in patients treated with Actemra than in placebo.

Actemra subcutaneous is also used for the treatment of moderate to severely active rheumatoid arthritis. Intravenous Actemra was also previously approved for the treatment of moderate to severely active rheumatoid arthritis, systemic juvenile idiopathic arthritis and polyarticular juvenile idiopathic arthritis. Intravenous administration is not approved for giant cell arteritis.