The FDA has granted breakthrough therapy designation to tocilizumab (Actemra, Genentech) for the treatment of systemic sclerosis, a rare and potentially life-threatening disease with no FDA-approved therapy.
FDA has granted breakthrough therapy designation to tocilizumab (Actemra, Genentech) for the treatment of systemic sclerosis, or scleroderma, a rare and potentially life-threatening disease with no FDA-approved therapy.
The breakthrough therapy designation was granted based on 48-week data from the phase 2 faSScinate study, presented as an oral presentation in Rome at EULAR 2015.
The aspects of systemic sclerosis where tocilizumab showed encouraging results in the phase 2 faSScinate trial were excessive skin thickening, deterioration of lung function and patient-reported outcome measures, according to Elena Hitraya, MD, PhD, group medical director, Genentech Immunology.
“Skin thickening and deterioration of lung function are important causes of increased morbidity and mortality in systemic sclerosis,” Dr Hitraya said. “By impacting these important aspects of disease [tocilizumab] may reduce disability, reduce the associated costs to the healthcare system as well as improve health-related quality of life. An effective disease-modifying drug could also reduce the 30% 10-year mortality associated with systemic sclerosis.”
Breakthrough designation represents a recognition by the FDA that the systemic sclerosis population we are studying with [tocilizumab] is an important area of unmet medical need, according to Jeff Siegel, MD, senior group medical director, Genentech Immunology. “The designation provides recognition that FDA believes the data are strong and that [tocilizumab] may provide benefit for a serious aspect of systemic sclerosis,” Dr Siegel said.
Systemic sclerosis is a debilitating rare, chronic autoimmune disease characterized by blood vessel abnormalities and scarring in the skin, joints and multiple internal organs. It affects 75,000 to 100,000 people in the United States and has the highest mortality of any autoimmune rheumatic disease, according to Dr Hitraya. It affects predominantly women and most commonly affects patients aged 30 to 50 years.
“Skin involvement can be debilitating by affecting patients’ ability to work or even to perform ordinary tasks of daily living like dressing and bathing,” she said. “Lung involvement is one of the most common forms of internal organ system involvement and represents one of the main causes of increased mortality.”