FDA Grants Breakthrough Therapy Designation for Actemra in Systemic Sclerosis


Genentech, a member of the Roche Group, was granted FDA Breakthrough Therapy Designation status for Actemra® (tocilizumab) for systemic sclerosis, also known as scleroderma. This designation is intended to expedite the development and review of medicines with early signals of potential clinical benefit in serious diseases and to help ensure patients have access to them as soon as possible.  Genentech has also initiated a Phase III study in systemic sclerosis (NCT02453256).

“The development of Actemra in systemic sclerosis represents our commitment to severe rheumatic diseases,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development.  “This Breakthrough Therapy Designation underscores the unmet need in systemic sclerosis and the potential of Actemra to help patients with this debilitating autoimmune disorder.”

Systemic sclerosis, a rare, chronic disorder, is characterized by blood vessel abnormalities, as well as degenerative changes and scarring in the skin, joints, and internal organs. The disease affects approximately 75,000 to 100,000 people in the United States. More than 75 percent of systemic sclerosis patients are women, primarily aged 30 to 50.1 Systemic sclerosis has the highest mortality of any autoimmune rheumatic disease.

There is currently no FDA-approved treatment for systemic sclerosis. There are treatments for some aspects of this disease, but none are used to stop or reverse the key symptom of skin thickening and hardening.

The Breakthrough Therapy Designation for Actemra was granted based on data from the Phase II faSScinate study. Forty-eight week data was presented as an oral presentation in Rome at EULAR 2015 (abstract number: OP0054; presentation date: Thursday, June 11, 2015). While the 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. In this second part of the study, there was continued improvement in skin thickening between weeks 24 and 48.5 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 II results and the unmet need in patients with systemic sclerosis, Genentech initiated a multicenter, randomized, double-blind, placebo-controlled Phase III trial (NCT02453256).



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