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Tocilizumab (Actemra/RoActemra) Receives FDA ‘Breakthrough Therapy’ Designation for Treatment of Systemic Sclerosis
The FDA has granted “breakthrough therapy” status to tocilizumab (Actemra/RoActemra, Roche) for the treatment of patients with systemic sclerosis (SSc). This designation is designed to expedite the development and review of medications intended to treat serious diseases, and to help ensure that patients have access to them as soon as possible.
SSc is a rare, chronic disorder characterized by blood-vessel abnormalities as well as by degenerative changes and scarring in the skin, joints, and internal organs. The disease affects approximately 2.5 million people worldwide and has the highest mortality of any rheumatic disorder.
The “breakthrough therapy” designation was granted to tocilizumab based on data from the mid-stage faSScinate trial. While the study’s primary endpoint of improvement in skin thickening at 24 weeks, as assessed by the Rodnan skin score, was not met, a “meaningful trend” was observed, according to Roche. Continued improvement in skin thickening was observed between weeks 24 and 48. The extent and severity of skin thickness correlates to disease worsening, increased disability, and decreased survival.
Based on the results of this phase II trial, Roche has initiated a global, phase III, randomized, double-blind, placebo-controlled study of tocilizumab in patients with SSc.
Tocilizumab is the first anti–interleukin-6 receptor (IL-6R) biologic agent approved in intravenous and subcutaneous formulations for the treatment of adult patients with moderate-to-severe active rheumatoid arthritis (RA). The treatment 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) RA management guidelines, tocilizumab is identified as the only biologic that has been demonstrated to be superior as monotherapy over MTX or other conventional disease-modifying antirheumatic drugs (DMARDs).
Clinical research has shown that effective treatment during the early phase of RA may prevent irreversible damage to joints and long-term disability. In patients who were diagnosed less than 1 year before study enrollment with no history of a previous disease-modifying therapy, tocilizumab nearly doubled sustained remission (SR) rates, with comparable results when used as monotherapy and as part of a combination. SR rates were 84% for tocilizumab monotherapy, 86% for tocilizumab plus MTX, and 44% for MTX alone. The median time to SR was 9.9 weeks for tocilizumab plus MTX and 12.7 weeks for tocilizumab monotherapy.
The phase III TENDER trial demonstrated that 97% of patients with systemic juvenile idiopathic arthritis (JIA) achieved 30% improvement in their disease symptoms (JIA ACR30) and that 64% achieved 90% improvement (JIA ACR90). The efficacy of tocilizumab was maintained through week 260 (4.9 years).
Tocilizumab is the only agent approved for the treatment of both systemic and polyarticular JIA in patients 2 years of age and older.
Source: Roche; June 10, 2015.