Giant Cell Arteritis Drug Receives Breakthrough Designation

The FDA grants autoimmune condition drug additional Breakthrough Therapy Designation.

Roche recently announced that the FDA granted Breakthrough Therapy Designation to Actemra/RoActemra (tocilizumab), an anti-IL-6 receptor biologic drug, in patients with giant cell arteritis.

Giant cell arteritis is a chronic autoimmune condition characterized by inflammation of large and medium-sized arteries in the head, as well as in the aorta and its branches, according to a press release from Roche.

Patients with giant cell arteritis are typically over 50-years-old. Women are more likely to be affected by the disease than men. If left untreated, it can cause blindness, aortic aneurysm, and stroke; however, treatment options are limited to high-doses of steroids to prevent vision loss.

These treatments often come with serious side effects, and do not maintain disease control on a long-term scale. This latest Breakthrough Therapy Designation is the second received for Actemra/RoActemra, and comes from the positive results announced from the phase 3 GiACTA study.

The study evaluated the efficacy and safety of Actemra in 251 patients with giant cell arteritis. Researchers combined Actemra with a glucocorticoid regimen for the first 6 months of treatment.

They discovered that patients receiving Actemra were able to sustain remission through 1 year compared with a steroid-only regimen.

Certain severe side effects can occur with treatment, including stomach tears, low neutrophil and platelet counts, increases in liver function test levels, increases in blood cholesterol levels, an increase in cancer risk, hepatitis B infection, serious allergic reactions, and nervous system problems. Other more common side effects, such as upper respiratory tract infections, headache, hypertension, injection site reactions, and diarrhea, were also reported.

The drug is currently approved in intravenous and subcutaneous formulations, and can be used with methotrexate in patients who are intolerant or who failed to respond to anti-rheumatic treatments, according to Roche.

The drug has been approved in 115 countries to date. It is also in an ongoing phase 3 clinical trial for patients with systemic sclerosis.

“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. “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.”