Tocilizumab approved for giant cell arteritis

Genentech today announced the approval of tocilizumab (Actemra) for the treatment of giant cell arteritis (GCA) in adults.

“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, MD, chief medical officer at Genentech. “With no new treatments in more than 50 years, this approval could be transformational for people with GCA and for their physicians.”

This is the sixth approved indication for tocilizumab, a novel humanized interleukin-6 (IL-6) receptor antagonist. Launched in 2010, the drug is also used to treat moderate-to-severe rheumatoid arthritis and juvenile idiopathic arthritis (JIA). Tocilizumab had previously received breakthrough therapy designation and priority review status from the FDA. 

Results from a phase 3 randomized, double-masked, placebo-controlled trial (GiACTA) showed tocilizumab outperformed steroid-only treatment in patients with GCA. The multicenter study was conducted in 251 patients across 76 sites in 14 countries.

At 1 year, tocilizumab, initially combined with a 6-month glucocorticoid regimen, more effectively sustained remission compared with a steroid regimen alone. Additionally, investigators also observed more visual complications in the placebo groups at the time of flare.

“The results of the GiACTA study mark a huge step forward for patients with GCA. For more than 65 years, no medication besides glucocorticoids has been proven in a convincing way to be effective,” said John H. Stone, MD, MPH, GiACTA’s lead investigator and professor of medicine at Harvard Medical School. “This rigorous, randomized and carefully blinded study addresses one of the greatest needs of physicians who treat GCA – finding an alternative to prolonged periods of steroid therapy.”