Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Kevzara^® (sarilumab) for the treatment of polymyalgia rheumatica (PMR), an inflammatory rheumatic disease, in adult patients who have had an inadequate response to corticosteroids or who cannot tolerate corticosteroid taper.

"Polymyalgia rheumatica can be an incapacitating disease, causing painful disease flares in multiple parts of the body that leave people fatigued and unable to fully perform everyday activities. Corticosteroids have been the primary treatment to date, but many patients do not adequately respond to steroids or cannot be tapered off steroids, which puts such patients at risk of complications from long-term steroid therapy,” said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron. “With the approval of Kevzara for polymyalgia rheumatica, patients now have an FDA-approved treatment to help offer relief from the disabling symptoms of this disease and long-term dependance on steroids.”

The FDA evaluated the Kevzara application for PMR under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Kevzara was previously approved for the treatment of moderately-to-severely active rheumatoid arthritis (RA) in adult patients who have had an inadequate response or intolerance to one or more disease-modifying antirheumatic drugs.

PMR often initially presents with pain and stiffness around the neck, shoulder and hip area and symptoms include fatigue, low-grade fever and weight loss. Patients often experience flares during tapering of, or relapse after discontinuation of corticosteroid (CS) treatment. Patients with PMR report difficulty in carrying out everyday functions such as getting out of bed, standing up from a chair, or lifting their arms. PMR generally affects people who are 50 years and older.

“Until now, people living with polymyalgia rheumatica have had limited treatment options for this serious rheumatic condition, which causes significant pain and discomfort,” said Bill Sibold, Executive Vice President, Head, Specialty Care at Sanofi. “The approval of Kevzara as the first and only biologic for polymyalgia rheumatica is a new option for patients and the healthcare professionals who treat them.”

The FDA approval for this additional indication for Kevzara is based on results from the SAPHYR Phase 3 randomized clinical trial in patients with steroid-resistant active PMR, who flared on ≥7.5 mg/day prednisone or equivalent during taper. In the trial, patients were randomized to receive either Kevzara 200 mg every two weeks along with a 14-week taper of CS (n=60; 1 patient randomized but not treated) or placebo every two weeks along with a 52-week CS taper (n=58). At 52 weeks, the trial met its primary endpoint with 28% of Kevzara-treated patients achieving sustained remission compared to 10% for placebo (p=0.0193). Sustained remission was defined as being in disease remission by week 12, absence of disease flare, C-reactive protein normalization from weeks 12 to 52, and adherence to the CS taper protocol from weeks 12 to 52.

A sensitivity analysis removing acute phase reactants (measures of ongoing inflammation) maintained significance (proportion difference for Kevzara vs. placebo: 18%; 95% confidence interval: 3.1 to 32.6) and confirmed the primary outcome. In addition, an analysis of a secondary endpoint showed that the median cumulative CS dose was 777 mg for Kevzara, compared to 2044 mg for placebo.

The common adverse reactions occurring in ≥5% of patients treated with Kevzara (n=59) were neutropenia (15%), leukopenia (7%), constipation (7%), rash pruritic (5%), myalgia (7%), fatigue (5%), and injection site pruritus (5%). Serious adverse reaction of neutropenia occurred in 2 patients (3%) in the Kevzara group compared to none in the placebo group (n=58). In both cases of neutropenia, the participants had a neutrophil count less than 500 per mm³ without any infections; neutropenia resolved following permanent discontinuation of study drug. The most common adverse reactions that resulted in permanent discontinuation of therapy with Kevzara were neutropenia occurring in 3 patients (5%) and infection in 3 separate patients (5%), including COVID-19 (n=1), intervertebral discitis (n=1), and pneumonia (n=1).

Regeneron and Sanofi are committed to helping patients in the U.S. who are prescribed Kevzara gain access to the medicine and receive the support they may need. KevzaraConnect^®, a comprehensive and specialized program that provides support services to patients throughout every step of the treatment process, can help eligible patients who are uninsured, lack coverage, or need copay assistance