Breakthrough Designation Granted to Autoimmune Disease Drug

The FDA has granted Breakthrough Therapy Designation status to Roche’s Actemra/RoActemra, also known as tocilizumab, for the treatment of giant cell arteritis (GCA). The chronic, life-threatening autoimmune condition is caused by the inflammation of large and medium-sized arteries, typically in the head, but also in the aorta and its branches.

Actemra/RoActemra is an anti—IL-6 receptor biologic that is available in both intravenous and subcutaneous formulations for the treatment of adult patients with moderate to severe active rheumatoid arthritis. It can also be used with methotrexate to treat adults who have failed to respond to other antirheumatic medications.

In June 2016, Roche announced the positive outcomes of a phase 3 study evaluating Actemra/RoActemra in patients with GCA. When used in combination with a 6-month steroid regimen, patients more effectively sustained remission through 1 year compared with those who were on a 6- or 12-month steroid-only regimen.

“The FDA Breakthrough Therapy designation for GCA underscores our continued commitment to explore Actemra/RoActemra in autoimmune diseases with significant unmet need,” said Sandra Horning, MD, Chief Medical Officer and head of Global Product Development at Roche. “We are looking forward to working with the FDA in the hope of making Actemra/RoActemra available to people with GCA, a condition for which there has been no approved treatment in more than 50 years.”

Breakthrough designation expedites the development and review of a drug that can potentially provide clinical benefits for certain diseases. This is the second Breakthrough Therapy Designation granted for Actemra/RoActemra.