The Lancet published a study showing that treatment with Actemra (tocilizumab) resulted in a reduction in skin thickness and an improvement in lung function in patients with systemic sclerosis (SSc). SSc is a disabling autoimmune disease that results in thickening and scarring of the skin, abnormalities in respiratory function, and potentially fatal damage to internal organs. This study is the first placebo-controlled Phase II trial of Actemra for the treatment of SSc.
The disease has the highest case fatality among rheumatic diseases, with a cumulative survival of 75 percent at 5 years from diagnosis. There is currently no FDA-approved treatment for the disease.
“This study supports the important role of Actemra in the treatment of progressive systemic sclerosis,” said lead investigator Dr. Dinesh Khanna, MD, MS, Professor of Internal Medicine in the Division of Rheumatology and director of the University of Michigan Scleroderma Program. “After 48 weeks of treatment, patients treated with Actemra exhibited a greater decrease in skin sclerosis than patients on placebo and less decline in forced vital capacity. These results, along with the fact there is no approved disease-modifying treatment for systemic sclerosis, reinforces the need for a Phase III study for Actemra for the treatment of systemic sclerosis. Phase III is an international study that is currently recruiting. More information can be found at NCT02453256“.
Additional Results Showed:
- Researchers recorded a clinically meaningful decline in mRSS over 48 weeks in the Actemra group compared with the placebo group.
- After 48 weeks of treatment, the safety profile was consistent with the known complications of systemic sclerosis and the safety profile for Actemra.
- Tocilizumab-treated patients had less decline in lung function compared to patients on placebo.
“The FDA granted Breakthrough Therapy Designation to Actemra for systemic sclerosis in 2015,” said Jeffrey Siegel, MD, Senior Group Medical Director of Immunology at Genentech. “We hope to confirm with the Phase III trial that treatment with Actemra results in improvement in skin thickening over time and preservation of lung function.”
Additional Study Information
Eighty seven patients with progressive SSc were enrolled in the phase II, randomized, double-blind, placebo-controlled faSScinate study. Patients across Canada, France, Germany, the UK, and the USA were randomly assigned to either weekly subcutaneous tocilizumab 162 mg (n=44) or placebo (n=43) for 48 weeks, followed by open-label weekly tocilizumab for 48 weeks. The study did not meet its primary endpoint, which was the difference in mean change from baseline in mRSS at 24 weeks.
Article: Safety and efficacy of subcutaneous tocilizumab in adults with systemic sclerosis (faSScinate): a phase 2, randomised, controlled trial, Prof Dinesh Khanna, MD, Prof Christopher P Denton, PhD, Angelika Jahreis, MD, Prof Jacob M van Laar, MD, Tracy M Frech, MD, Marina E Anderson, FRCP, Murray Baron, MD, Lorinda Chung, MD, Gerhard Fierlbeck, MD, Santhanam Lakshminarayanan, MD, Prof Yannick Allanore, PhD, Janet E Pope, MD, Prof Gabriela Riemekasten, PhD, Prof Virginia Steen, MD, Prof Ulf Müller-Ladner, MD, Robert Lafyatis, MD, Giuseppina Stifano, MD, Helen Spotswood, PhD, Haiyin Chen-Harris, PhD, Sebastian Dziadek, PhD, Alyssa Morimoto, PhD, Thierry Sornasse, PhD, Jeffrey Siegel, MD, Prof Daniel E Furst, MD, The Lancet, doi: 10.1016/S0140-6736(16)00232-4, published online 5 May 2016.