FDA Approves First Drug to Specifically Treat Giant Cell Arteritis

Agency expands Actemra label

The FDA has expanded the approved use of subcutaneous tocilizumab (Actemra, Roche) to include adults with giant cell arteritis. This new indication provides the first FDA-approved therapy that is specific to this type of vasculitis.

The efficacy and safety of tocilizumab were evaluated in a double-blind, placebo-controlled study involving 251 patients with giant cell arteritis. The primary efficacy endpoint was the proportion of patients achieving sustained remission from week 12 through week 52. Sustained remission was defined as the absence of symptoms of giant cell arteritis; the normalization of inflammatory laboratory tests; and tapering the use of the steroid drug prednisone. A greater proportion of patients receiving subcutaneous tocilizumab with standardized prednisone regimens achieved sustained remission from week 12 through week 52 compared with patients receiving placebo with standardized prednisone regimens. In addition, the cumulative prednisone dose was lower in patients treated with tocilizumab compared with those given placebo.

The labeling for tocilizumab includes a boxed warning regarding serious infections. Patients treated with tocilizumab who develop a serious infection should stop treatment until the infection is controlled. Live vaccines should be avoided during treatment with tocilizumab. Tocilizumab should be used with caution in patients with an increased risk of gastrointestinal perforation. Hypersensitivity reactions, including anaphylaxis and death, have occurred. Laboratory monitoring is recommended because of the potential consequences of treatment-related changes in neutrophils, platelets, lipids, and liver-function tests.

Subcutaneous tocilizumab was previously approved for the treatment of patients with moderately to severely active rheumatoid arthritis (RA). Intravenous tocilizumab was also approved for the treatment of patients with moderately to severely active RA as well as systemic juvenile idiopathic arthritis and polyarticular juvenile idiopathic arthritis. Intravenous tocilizumab is not approved for patients with giant cell arteritis.

Source: FDA; May 22, 2017.