Tocilizumab Gets FDA Priority Review for Giant Cell Arteritis

The FDA gave tocilizumab priority designation for treating adults with GCA.

The US Food and Drug Administration (FDA) has accepted Genentech’s Supplemental Biologics License Application (sBLA) and granted priority for tocilizumab (Actemra/Genentech) to treat adults with giant cell arteritis (GCA) — a form of vasculitis.

The designation was based on the positive outcome from a phase 3 GiACTA study (52 weeks) including 251 patients that assessed tocilizumab’s use for patients with GCA.

Results showed that compared with patients who were only given a six or 12-month steroid (glucocorticoid) taper regimen, the group given Actemra combined with a six-month glucocorticoid regimen had more effectively maintained remission through one year.

The chronic autoimmune condition typically affects adults over the age of 50 and had been reported to cause severe headaches and jaw pain, and if left untreated can lead to vision loss, aortic aneurysm, and stroke.

According to John Stone, MD, of Massachusetts General Hospital, and GiACTA’s lead investigator, “Giant cell arteritis is a disease that has huge unmet need ever since cortisone was invented 67 years ago.” And, there had not been any new therapies for GCA in more than 50 years.

At the American College of Rheumatology (ACR) 2016 Annual Meeting in Washington, DC, Stone explained to MD Magazine in a video interview that the only existing treatment, glucocorticoids (prednisone), while effective, is associated with a host of side effects, making it difficult to maintain patients on prednisone for a long period of time. As such, the team embarked on the GiACTA study, to test their hypothesis and demonstrate that tocilizumab possessed a powerful “steroid-sparing” effect.

As GCA patients often are absorbing the toxicity of the glucocorticoid, the researchers highlight the importance of using both medicines together and eventually taper the steroids off as quickly as possible. The authors concluded in a news release, “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.