Roche has received 14 breakthrough therapy designations from the U.S. Food and Drug Administration since 2013 and this is the second time that the status has been awarded to Actemra.
The idea behind breakthrough designations is to facilitate the development of medicines with immediate evidence of potential positive clinical results in relation to serious diseases, assuring patients get the best access to new medical treatments.
"The FDA Breakthrough Therapy designation for giant cell arteritis underscores our continued commitment to explore Actemra/RoActemra in autoimmune diseases with significant unmet need," said Sandra Horning, Roche's chief medical officer.
Giant cell arteritis (also known as GCA, or temporal arteritis or TA) is an autoimmune condition with worldwide impact, generally affecting those who are above 50 years old, and especially women. It is part of the larger spectrum of large-vessel vasculitis conditions and usually affects the aorta as well as the aortic branches. Most of the documented cases of GCA involve dysfunctions of the temporal arteries.
If not detected on time, the condition can produce severe consequence on the physiological functions of the patients. Should the condition be untreated, it can lead to numerous consequences, some of which are life-threatening. Among the most severe of these are blindness, stroke or aortic aneurysm.
The disease is usually not diagnosed during the early stages, because the symptoms are varied and different from one another. From loss of vision to fever and headache, the wide array of symptoms makes treatment occur in the later stages.
The most common treatment of the later stages of GCA involves high doses of steroids. While efficient in emergency cases, they fail to guarantee long-term effects, and usually involve severe side effects. The wide array of symptoms and the complex manifestations of the disease make it very difficult to diagnose early stages of GCA, which leads to a large use of steroids in advanced-stage treatments.
As Actemra/RoActemra is the only approved treatment for both intravenous and subcutaneous forms of GCA, patients with moderate and higher rheumatoid arthritis will be able to use it in their conventional medication.
The subcutaneous development of the medicine involved two Phase III clinical studies, also enrolling almost 2,000 people from 33 countries suffering from rheumatoid arthritis. The intravenous formula is used for treating polyarticular juvenile idiopathic arthritis. The company's treatments are approved in no less than 115 countries around the globe.
Actemra is co-developed by Chugai Pharmaceutical, a Tokyo-based company specialized in producing biotechnology treatments. A lead member of the Roche Group, the research-based organization is striving to create new products and methods to meet the most recent medical needs worldwide, with a focus on oncology.