The US Food and Drug Administration (FDA) has accepted the Supplemental Biologics License Application (sBLA) for tocilizumab (Actemra®, Genentech, South San Francisco, CA) for the treatment of giant cell arteritis (GCA). The regulator also gave tocilizumab Priority Review Designation for treatment of GCA, which means the FDA plans a review decision within 6 months rather than the standard 10-month review.1
The FDA describes Priority Review as “direct[ing] overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.”
In October 2016, the FDA granted a Breakthrough Therapy Designation to tocilizumab for the treatment of several autoimmune diseases, as reported by Rheumatology Advisor. It was the first approved treatment for GCA in more than half a century.
Tocilizumab is a humanized interleukin-6 receptor antagonist that has been approved to treat adult patients with moderate to severe active rheumatoid arthritis and GCA, as well as polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis in patients aged 2 and older.
The FDA’s decision to give tocilizumab priority review was based on positive results from the GiACTA study (ClinicalTrials.gov Identifier: NCT01791153), a phase 3, double-blind, randomized controlled trial that evaluated the efficacy of tocilizumab for treatment of GCA.2
“GiACTA represents the first real breakthrough in the treatment of giant cell arteritis in 67 years, since the invention of cortisone,” said the study’s lead investigator, John H. Stone, MD, MPH, a professor of medicine at Harvard Medical School and director of clinical rheumatology and Edward Fox Chair in Medicine at the Massachusetts General Hospital.
“The most surprising finding from the trial, I think, was how poorly prednisone alone did in leading to sustained remissions,” Dr Stone previously told Rheumatology Advisor when discussing the trial results.
“At one year in the group of patients in the trial who received prednisone tapered over a full year, only 18% of those patients achieved the primary outcome [of sustained GCA remission],” he added. “In comparison, both of the tocilizumab groups achieved the primary outcome at a much higher rate: 56% for those who received weekly tocilizumab and 53% who received tocilizumab every other week.”
Dr Stone concluded that the study results “mark a huge step forward” for patients with GCA and their treatment options.
“In more than 50 years, no medication other than glucocorticoids has ever been shown in a convincing way to be effective.”
The GiACTA clinical trial was funded by Hoffmann-La Roche. Genentech is a member of the Roche Group.
FDA grants priority review for Genentech’s Actemra® (tocilizumab) Supplemental Biologics License Application for giant cell arteritis, a form of vasculitis [news release]. Published January 23, 2017. Accessed January 24, 2017.
Stone JH, Tuckwell K, Dimonaco S, et al. Efficacy and safety of tocilizumab in patients with giant cell arteritis: primary and secondary outcomes from a phase 3, randomized, double-blind, placebo-controlled trial [abstract]. Arthritis Rheumatol. 2016;68(suppl 10).