Genentech, a member of the Roche Group, announced that the FDA has approved Actemra (tocilizumab) subcutaneous injection for the treatment of giant cell arteritis (GCA), a chronic and severe autoimmune condition. Actemra is the first therapy approved by the FDA for the treatment of adult patients with GCA. This is the sixth FDA approval for Actemra since the medicine was launched in 2010.

“Today’s FDA decision means people living with giant cell arteritis will, for the first time, have an FDA-approved treatment option for this debilitating disease,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “With no new treatments in more than 50 years, this approval could be transformational for people with GCA and for their physicians.”

The approval is based on the positive outcome of the phase III GiACTA study evaluating Actemra in patients with GCA. The results showed that Actemra, initially combined with a six-month steroid (glucocorticoid) regimen, more effectively sustained remission through 52 weeks (56 percent in the Actemra weekly group and 53.1% in the Actemra bi-weekly group) compared to placebo combined with a 26-week steroid taper (14%) and placebo combined with a 52-week steroid taper (17.6%).

GiACTA (NCT01791153) is a phase III, global, randomized, double-blind, placebo-controlled trial investigating the efficacy and safety of Actemra as a novel treatment for GCA. It is the first successful clinical trial conducted in GCA and the first to use blinded, variable-dose, variable-duration steroid regimens. The multicenter study was conducted in 251 patients across 76 sites in 14 countries. The primary endpoint was evaluated at 52 weeks.

GCA, also known as temporal arteritis (TA), affects an estimated 228,000 people over the age of 50 in the U.S., and the disease is two to three times more likely to affect women than men. GCA is often difficult to diagnose because of the wide and variable spectrum of signs and symptoms. GCA 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 GCA has been limited to high-dose steroids that play a role as an effective ‘emergency’ treatment option to prevent damage such as vision loss. Due to the variability of symptoms, complexity of the disease and disease complications, people with GCA are often seen by several physicians including rheumatologists, ophthalmologists and neurologists.

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 (RA) who have used one or more disease-modifying antirheumatic drugs (DMARDs), such as methotrexate (MTX), that did not provide enough relief. The extensive Actemra RA IV clinical development program included five phase III clinical studies and enrolled more than 4,000 people with RA in 41 countries. The Actemra RA subcutaneous clinical development program included two phase III clinical studies and enrolled more than 1,800 people with RA in 33 countries. In addition, Actemra is also used as an IV formulation for patients with active polyarticular juvenile idiopathic arthritis (PJIA) or systemic juvenile idiopathic arthritis (SJIA) two years of age and older. Actemra is not approved for subcutaneous use in people with PJIA or SJIA. It is not known if Actemra is safe and effective in children with PJIA or SJIA under two years of age or in children with conditions other than PJIA or SJIA.