Tocilizumab (Actemra) is the first drug approved to treat giant cell arteritis.
The FDA recently expanded the approval of tocilizumab (Actemra) to treat patients with giant cell arteritis (GCA). The new approval makes tocilizumab the first drug approved to treat the condition, according to a press release.
“We expedited the development and review of this application because this drug fulfills a critical need for patients with this serious disease who had limited treatment options,” said Badrul Chowdhury, MD, PhD, director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research.
GCA is a type of vasculitis characterized by inflammation of blood vessels, which causes arteries to narrow or become irregular and results in inadequate blood flow.
In patients with GCA, the affected blood vessels typically occur in the head, especially in the temporal arteries, according to the release. However, patients may still experience inflammation in large arteries, such as the aorta.
Currently, patients are treated with high doses of corticosteroids that are tapered over time, the FDA reported.
The new approval was based on positive findings from a clinical trial exploring the safety and efficacy of subcutaneous tocilizumab. Included in the study were 251 patients with GCA who were randomized to receive tocilizumab or placebo. The primary efficacy endpoint of the trail was the number of patients achieving remission from week 12 to week 52.
Sustained remission was defined as the absence of GCA symptoms, normalized inflammatory laboratory tests, and tapered use of prednisone, according to the release.
The investigators discovered that a larger portion of patients treated with subcutaneous tocilizumab plus standard prednisone achieved sustained remission compared with patients treated with placebo plus prednisone, the FDA reported. Additionally, prednisone use was lower in patients treated with tocilizumab compared with placebo patients.
The overall safety profile of tocilizumab in patients with GCA was similar to its known profile. Tocilizumab does carry a boxed warning over risks of serious infections. The FDA advises that patients who develop a serious infection should stop treatment until the infection is eliminated. Live vaccines should also be avoided during treatment and the drug should be used cautiously in patients with increased risk of gastrointestinal perforation, according to the FDA.
The FDA noted that tocilizumab can lead to anaphylaxis and death in some patients. Additionally, patients should undergo laboratory monitoring due to potential changes in neutrophil, platelet, lipid, and liver function tests.
The FDA previously granted tocilizumab breakthrough therapy and priority review for the treatment of GCA.
Subcutaneous tocilizumab was previously approved to treat moderate-to-severe rheumatoid arthritis, while intravenous tocilizumab has been approved to treat moderate-to-severe rheumatoid arthritis, systemic juvenile idiopathic arthritis, and polyarticular juvenile idiopathic arthritis, according to the FDA.
This new approval applies only to subcutaneous tocilizumab and not the intravenous formulation of the drug, according to the release.
“Today’s FDA decision means people living with giant cell arteritis will, for the first time, have an FDA-approved treatment option for this debilitating disease,” said Sandra Horning, MD, chief medical officer and head of Global Product Development, in a press release. “With no new treatments in more than 50 years, this approval could be transformational for people with GCA and for their physicians.”