Roche announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation (BTD) status to ACTEMRA®/RoACTEMRA® (tocilizumab) for systemic sclerosis (SSc). This designation is designed to expedite the development and review of medicines intended to treat serious diseases, and to help ensure patients have access to them as soon as possible.
SSc is a rare, chronic disorder characterised by blood vessel abnormalities, as well as degenerative changes and scarring in the skin, joints, and internal organs.The incidence of SSc is difficult to measure but is estimated to affect approximately 2.5 million people worldwide, and has the highest mortality of any rheumatic disease. FDA BTD status for ACTEMRA/RoACTEMRA was granted based on data from the Phase 2 faSScinate study. 48 week data from faSScinate will be presented as an oral presentation at EULAR 2015. While the primary endpoint of improvement in skin thickening at 24 weeks, as assessed by Rodnan skin score, was not met a meaningful trend was observed. In this second part of the study, there was continued improvement in skin thickening between weeks 24 and 48. The overall adverse event profile between both groups was comparable. The extent and severity of skin thickness correlates to disease worsening, increased disability and decreased survival. Based on these Phase 2 results and the unmet need in patients with SSc, for which there are no approved disease modifying therapeutic options, Roche initiated a global Phase 3 multicentre, randomised, double-blind, placebo-controlled stud.
ACTEMRA/RoACTEMRA is the first anti-IL-6 receptor biologic approved in intravenous (IV) and subcutaneous formulations, for the treatment of adult patients with moderate to severe active RA. ACTEMRA/RoACTEMRA can be used alone or with MTX in adults who are intolerant to, or have failed to respond to other anti-rheumatic medications. In the most recent update to the EULAR RA management guidelines, ACTEMRA/RoACTEMRA is highlighted as the only biologic that has been repeatedly demonstrated to be superior as a monotherapy over MTX or other conventional disease-modifying anti-rheumatic drugs (DMARDs).ACTEMRA/RoACTEMRA IV formulation is approved in most major countries for polyarticular juvenile idiopathic arthritis (pJIA) or systemic juvenile idiopathic arthritis (sJIA) in children two years of age and older.
