The FDA has granted a breakthrough therapy designation for tocilizumab (Actemra/RoActemra, Roche) for the treatment of patients with giant cell arteritis (GCA), a potentially life-threatening autoimmune condition. The disease is caused by inflammation of large and medium-sized arteries, most often in the head, but also in the aorta and its branches.
Breakthrough therapy status is intended to expedite the development and review of medications with early evidence of a potential clinical benefit in serious diseases.
In June, Roche announced positive outcomes of the phase 3 GiACTA study evaluating tocilizumab in people with GCA. The results showed that tocilizumab, initially combined with a six-month steroid (glucocorticoid) regimen, more effectively sustained remission through one year compared with a six- or 12-month steroid-only regimen in patients with GCA.
Tocilizumab is the only anti-interleukin (IL)-6 receptor biologic available in both intravenous and subcutaneous formulations for the treatment of adults with moderately to severely active rheumatoid arthritis (RA). Tocilizumab can be used alone or with methotrexate (MTX) in adults who are intolerant of or have failed to respond to other antirheumatic medications. In the most recent update to the European League Against Rheumatism (EULAR) guidelines for RA management, tocilizumab is highlighted as the only biologic that has been shown to be superior as a monotherapy compared with MTX or other conventional disease-modifying antirheumatic drugs (DMARDs).
In addition to GCA, tocilizumab is being investigated in a global phase 3, randomized, double-blind, placebo-controlled study in patients with systemic sclerosis (SSc). Tocilizumab was granted a breakthrough therapy designation for SSc in June 2015.
GCA––also known as temporal arteritis–– usually affects people older than 50 years of age, and the disease is two to three times more likely to affect women than men. GCA is often difficult to diagnose because of its broad spectrum of signs and symptoms. The disorder can cause severe headaches, jaw pain, and visual symptoms, and, if untreated, it can lead to blindness, aortic aneurysm, or stroke. Treatment for patients with GCA has been limited to the use of high-dose steroids, which play a role as an effective “emergency” treatment option to prevent damage, such as vision loss. Steroids, however, do not always maintain long-term disease control (flare-free remission) and are often associated with severe adverse effects.
Source: Roche; October 5, 2016.