The US Food and Drug Administration has assigned Roche’s Actemra breakthrough status for systemic sclerosis, potentially speeding up its development and review for this indication.
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, thought to affect around 2.5 million around the globe.
Actemra (tocilizumab; RoActemra in Europe) bagged its breakthrough status on the back of data from the Phase II faSScinate study. While the primary endpoint of improvement in skin thickening (a measure of disease progress) at 24 weeks was not met, a meaningful trend was observed, and there was continued improvement between weeks 24 and 48.5.
Given the data, and the high unmet need for patients with SSc, for which there are no approved disease modifying therapeutic options, Roche said is has now kicked off a global Phase III multicentre, randomised, double-blind, placebo-controlled study (NCT02453256) to further assess safety and efficacy of its drug.
Actemra won US approval back in 2010 as the first anti-IL-6 receptor biologic available in intravenous and subcutaneous formulations to treat adults with moderate-to-severe active rheumatoid arthritis.