FDA Approves Upadacitinib for Giant Cell Arteritis in Adults

The FDA has approved upadacitinib, a Janus kinase inhibitor, for the treatment of giant cell arteritis in adults-marking the first new approval for the condition since tocilizumab in 2017, according to a press release.

The approvals are supported by results of the pivotal Phase 3 SELECT-GCA clinical trial, which met the primary endpoint of sustained remission (46.4% of patients receiving RINVOQ 15 mg in combination with a 26-week steroid taper regimen achieved sustained remission from week 12 to week 52, compared to 29.0% of patients receiving placebo in combination with a 52-week steroid taper regimen; p=0.002).

During the 52-week, placebo-controlled period, the safety profile of RINVOQ was generally consistent with that observed in other approved indications.

"This FDA approval will now provide an alternative treatment option that can offer patients with GCA the possibility of tapering off steroids and achieving sustained remission," said Roopal Thakkar, M.D., executive vice president, research and development, chief scientific officer, AbbVie. "With this new indication for RINVOQ, we are underscoring AbbVie's commitment to exploring how we can identify and address unmet needs for patients with immune-mediated diseases."

GCA is an autoimmune disease that causes inflammation of the temporal and other cranial arteries, the aorta, and other large and medium arteries. If left untreated, the disease can lead to debilitating symptoms and potentially severe outcomes, such as blindness, aortic aneurysm, or stroke.2 Caucasian women over the age of 50 – most commonly between the ages of 70 and 80 years – have the highest risk of developing GCA. Although women are more likely than men to develop GCA, research suggests that men are more likely to have ocular manifestations with their disease.

"Glucocorticoids remain a mainstay of treatment of GCA but lead to substantial drug-associated toxicities. Additionally, relapse remains common for patients with this disease," said Peter A. Merkel, M.D., MPH, chief of rheumatology at the University of Pennsylvania, Philadelphia, and SELECT-GCA trial investigator. "We now have a new option to treat GCA. The results of this clinical trial show that upadacitinib offers patients the chance to reach sustained remission."

RINVOQ SAFETY CONSIDERATIONS

• RINVOQ may cause serious side effects, including:

• Serious infections. RINVOQ can lower ability to fight infections. Serious infections, some fatal, occurred, including tuberculosis (TB) and infections caused by bacteria, fungi, or viruses.

• Increased risk of death in people age 50+ with at least 1 heart disease risk factor.

• Cancer and immune system problems. Increased risk of some cancers, including lymphoma and skin. Current or past smokers have higher risk for lymphoma and lung cancer.

• Increased risk of major cardiovascular events such as heart attack, stroke, or death in people 50+ with at least 1 heart disease risk factor, especially in current or past smokers.

• Blood clots, some fatal, in veins of the legs or lungs and arteries. This occurred more often in people 50+ with at least 1 heart disease risk factor.

• Serious allergic reactions. Do not take if allergic to RINVOQ or its ingredients.

• Tears in the stomach or intestines; changes in certain laboratory test results.

• Sustained remission was defined as having an absence of GCA signs and symptoms from week 12 through week 52 and adherence to the protocol-defined corticosteroid taper regimen.

About SELECT-GCA

SELECT-GCA (M16-852) is a Phase 3, multicenter, randomized, double-blind placebo-controlled study designed to evaluate the safety and efficacy of upadacitinib (RINVOQ) in 428 patients with GCA. The study consists of two periods. The first period, previously reported in April 2024, evaluated the efficacy of upadacitinib in combination with a 26-week corticosteroid taper regimen compared to placebo in combination with a 52-week corticosteroid taper regimen. In addition, the study assessed the safety and tolerability of upadacitinib in these patients. The second period will evaluate the safety and efficacy of continuing versus withdrawing upadacitinib in maintaining remission in participants who achieved sustained remission in the first period. 

About Giant Cell Arteritis

Giant cell arteritis (GCA), also known as temporal arteritis, is an autoimmune disease of medium and large arteries, characterized by granulomatous inflammation of the three-layered vessel wall, which affects temporal and other cranial arteries as well as the aorta and other large arteries. GCA can cause headache, jaw pain, and changes in or loss of vision, including sudden and permanent loss of vision. It is the most common vasculitis affecting adults in Western countries.

About RINVOQ® (upadacitinib)

Discovered and developed by AbbVie scientists, RINVOQ is a JAK inhibitor that is being studied in several immune-mediated inflammatory diseases. In human leukocyte cellular assays, RINVOQ inhibited cytokine-induced STAT phosphorylation mediated by JAK1 and JAK1/JAK3 more potently than JAK2/JAK2 mediated STAT phosphorylation. The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness and safety is not currently known.

Upadacitinib (RINVOQ) is being studied in Phase 3 clinical trials for alopecia areata, hidradenitis suppurativa, Takayasu arteritis, systemic lupus erythematosus, and vitiligo.