Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s Supplemental Biologics License Application (sBLA) for Actemra® (tocilizumab) for the treatment of GCA, a chronic, potentially life-threatening autoimmune condition. The FDA also granted Priority Review Designation for Actemra for the treatment of GCA. The designation is based on the positive outcome of the Phase III GiACTA study evaluating Actemra in people with GCA. Results showed that Actemra, initially combined with a six-month steroid (glucocorticoid) regimen, more effectively sustained remission through one year compared to a six- or 12-month steroid taper regimen given alone in people with GCA.1
“This positive outcome in GCA, a condition for which there have been no new treatments in more than 50 years, demonstrates Genentech’s commitment to helping patients with unmet needs,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are pleased by the FDA’s decision to classify their review of the sBLA as priority. We will continue to work closely with the FDA to bring this investigational medicine to people with GCA as quickly as possible.”
Priority Review Designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment of a serious disease.
In October 2016, Genentech announced receiving Breakthrough Therapy Designation for the treatment of GCA with Actemra. Breakthrough designation is intended to expedite the development and review of medicines with early evidence of substantial clinical benefit in serious diseases and to help ensure that patients receive access to medicines as soon as possible.
About the GiACTA study
GiACTA (NCT01791153) is a Phase III, global, randomized, double-blind, placebo-controlled trial investigating the efficacy and safety of Actemra as a novel treatment for GCA. It is the largest clinical trial ever conducted in GCA and the first to use blinded, variable-dose, variable-duration steroid regimens. The multicenter study was conducted in 251 patients across 76 sites in 14 countries. The primary and key secondary endpoints were evaluated at 52 weeks.
About Giant Cell Arteritis
The prevalence of GCA has been estimated at over 200 per 100,000 persons in the U.S. over the age of 50, and the disease is two to three times more likely to affect women than men.2,3 GCA is often difficult to diagnose because of the wide and variable spectrum of signs and symptoms. GCA can cause severe headaches, jaw pain and visual symptoms and if left untreated, can lead to blindness, aortic aneurysm or stroke.2 Treatment to date for people with GCA has been limited to high-dose steroids that play a role as an effective ‘emergency’ treatment option to prevent damage such as vision loss. However, steroids are often unable to maintain long-term disease control (flare-free remission).3,4,5 Due to the variability of symptoms, complexity of the disease and disease complications, people with GCA are often seen by several physicians including rheumatologists, neurologists and ophthalmologists.
Actemra is the first humanized interleukin-6 (IL-6) receptor antagonist approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have used one or more disease-modifying antirheumatic drugs (DMARDs), such as methotrexate (MTX), that did not provide enough relief. The extensive Actemra RA IV clinical development program included five Phase III clinical studies and enrolled more than 4,000 people with RA in 41 countries. The Actemra RA subcutaneous clinical development program included two Phase III clinical studies and enrolled more than 1,800 people with RA in 33 countries. In addition, Actemra is also used as an IV formulation for patients with active polyarticular juvenile idiopathic arthritis (PJIA) or systemic juvenile idiopathic arthritis (SJIA) two years of age and older. Actemra is not approved for subcutaneous use in people with PJIA or SJIA.
Actemra is intended for use under the guidance of a healthcare practitioner.