Roche was recently granted breakthrough therapy designation (BTD) by the U.S. Food and Drug Administration (FDA) for its combined treatment ACTEMRA/RoACTEMRA (tocilizumab) for systemic sclerosis (SSc). The status was awarded based on successful phase 2 studies, which are being presented this week at the European League Against Rheumatism annual congress (EULAR 2015), in Rome, Italy.
The breakthrough therapy designation is granted by the FDA to accelerate the development and review processes of medication expected to be effective in the treatment of severe conditions, such as SSc, which is a rare and chronic disease that causes abnormalities in the blood vessels, degenerative alterations and scarring in the organs, skin and joints. In addition, the status is expected to enable patients to have access to new therapies faster, reducing the burden of the disease.
The FDA granted ACTEMRA/RoACTEMRA the designation based on results from the phase 2 faSScinate study, which will be presented at EULAR 2015. Despite the fact that during the 48-week the primary endpoint was not met, which was improvement of skin thickening at 24 weeks measured by Rodnan skin score, the investigators did report a meaningful trend. In a second part of the trial, patients’ skin thickening improved from weeks 24 to 48, while the overall adverse event profile was comparable in both groups.
The researchers concluded that extent and severity of skin thickness is related to worsening of the condition, as well as increased disability and decreased survival. Given the encouraging results of the phase 2 study, as well as severity of SSc, a disease for which there is currently no cure or disease modifying treatments approved, the company decided to begin its global phase 3 multi center, randomized, double-blind, placebo-controlled study.
“Close to 500,000 people worldwide have benefited from treatment with ACTEMRA/RoACTEMRA since initial approval over a decade ago,” stated the head of Global Product Development and chief medical officer at Roche, Sandra Horning, MD. “The breadth of our study results at EULAR underscores our commitment to helping people with debilitating autoimmune diseases. These new data further demonstrate the efficacy and safety of ACTEMRA/RoACTEMRA in multiple diseases.”
In addition to the data released on ACTEMRA/RoACTEMRA as a potential treatment for SSc, the company is also presenting at EULAR 2015 results from the U-ACT-EARLY study that assessed the therapy in patients with early rheumatoid arthritis, as well as from the TENDER study in patients with systemic juvenile idiopathic arthritis (sJIA).
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