FDA grants Breakthrough designation for Actemra in Systemic Sclerosis
Genentech, a member of the Roche Group, has announced that the FDA has granted Breakthrough Therapy designation status to 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 also has initiated a phase III study in systemic sclerosis (NCT02453256), a disease for which there are inadequate treatment options.
Systemic sclerosis is a rare, chronic disorder 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 U.S. More than 75% of systemic sclerosis patients are women, primarily aged 30 to 50. Systemic sclerosis has the highest mortality of any autoimmune rheumatic disease.
There currently is 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 will be presented as an oral presentation in Rome at EULAR 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. 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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