The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to tocilizumab (Actemra®, Genentech) for the treatment of multiple autoimmune diseases.
Actemra is a humanized interleukin-6 (IL-6) receptor antagonist. It has been approved to treat adult patients with moderate-to-severe active rheumatoid arthritis (RA), giant cell arthritis (GCA), polyarticular juvenile idiopathic arthritis (PJIA), and systemic juvenile idiopathic arthritis (SJIA) in patients aged 2 and older.
“The FDA Breakthrough Therapy Designation…underscores our continued commitment to explore Actemra in autoimmune diseases with significant unmet need,” said Sandra J. Horning, MD, chief medical officer and head of global product development at Genentech, Inc.
Actrema is the first approved treatment in more than 50 years for GCA, a chronic and potentially life-threatening disease typically found in persons age 50 or older. Symptoms include severe headaches, jaw pain, and visual symptoms that can lead to blindness, aortic aneurysm, and stroke. Past treatment has been limited to high-dose steroids, which do not always prevent flare-free remission.
Actrema use may cause serious side effects including gastric or intestinal perforation, hepatitis B infection, serious allergic reactions, multiple sclerosis, an increased risk of certain cancers, low absolute neutrophil counts, and thrombocytopenia.
Full data from the phase 3 GiACTA trial (ClinicalTrials.gov Identifier: NCT01791153), a multicenter, randomized-double-blind, placebo-controlled study evaluating the use of Actemra in patients with active GCA, will be presented at an upcoming medical meeting in 2016.
The primary outcome measure evaluated in GiACTA is the proportion of patients who maintain sustained remission 52-week post-randomization. Secondary outcome measures include time to GCA disease flare after clinical remission, reductions in total cumulative prednisone doses, and patient reported outcomes.
The NCT01791153 clinical trial is sponsored by Hoffman-LaRoche.
FDA grants breakthrough therapy designation for Genentech’s Actemra (tocilizumab) in giant cell arthritis, a form of vasculitis [news release]. http://www.businesswire.com/news/home/20161004006731/en/FDA-Grants-Breakthrough-Therapy-Designation-Genentech; Published October 5, 2016. Accessed October 5, 2016.