The FDA ( Food and Drug Administration ) has granted Breakthrough Therapy Designation status to Actemra ( Tocilizumab; in Europe: RoActemra ) for giant cell arteritis ( GCA ), a chronic, potentially life-threatening autoimmune condition. The disease is caused by inflammation of large and medium-sized arteries, most often in the head, but also in the aorta and its branches.
Breakthrough designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases and to help ensure that patients receive access to medicines as soon as possible. This is the second Breakthrough Therapy Designation for Actemra.
In June of this year, Genentech announced the positive outcome of the phase III GiACTA study evaluating Actemra in people with GCA. Results showed that Actemra, initially combined with a six-month steroid ( glucocorticoid ) regimen, more effectively sustained remission through one year compared to a 6- or 12-month steroid-only regimen in people with giant cell arteritis.
The prevalence of giant cell arteritis has been estimated at over 200 per 100,000 persons in the U.S. over the age of 50, and the disease is two to three times more likely to affect women than men.
Giant cell arteritis is often difficult to diagnose because of the wide and variable spectrum of signs and symptoms.
Giant cell arteritis can cause severe headaches, jaw pain and visual symptoms and if left untreated, can lead to blindness, aortic aneurysm or stroke.
Treatment to date for people with giant cell arteritis has been limited to high-dose steroids that play a role as an effective emergency treatment option to prevent damage such as vision loss. However, steroids do not always maintain long-term disease control ( flare-free remission ).
Actemra is the first humanized interleukin-6 ( IL-6 ) receptor antagonist approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have used one or more disease-modifying antirheumatic drugs ( DMARDs ), such as Methotrexate, that did not provide enough relief. ( Xagena )
Source: Genentech, 2016