FDA approves sarilumab as first biologic to treat polymyalgia rheumatica
Click Here to Manage Email Alerts
The FDA has approved sarilumab for the treatment of polymyalgia rheumatica in adults who have experienced an inadequate response to, or cannot taper, corticosteroids, according to a press release from Sanofi.
Sarilumab (Kevzara; Sanofi, Regeneron), an injectable interleukin-6 receptor blocker, is the first biologic to gain FDA approval for the disease.
“Polymyalgia rheumatica can be an incapacitating disease, causing painful disease flares in multiple parts of the bodies that leave people fatigued and unable to fully perform everyday activities,” George D. Yancopolous, MD, PhD, president and chief scientific officer at Regeneron, which is jointly developing sarilumab alongside Sanofi under a global collaboration agreement, said in the release.
“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,” he added. “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 dependence on steroids.”
According to Sanofi, the approval is based on results of the phase 3 SAPHYR study, in which three times more patients treated with sarilumab achieved sustained remission vs. placebo. The randomized controlled trial enrolled patients with steroid-resistant active polymyalgia rheumatica who experienced flare while receiving at least 7.5 mg of daily prednisone or equivalent during taper. Among these patients, 60 were randomly assigned to sarilumab 200 mg every 2 weeks with a 14-week corticosteroid taper, while 58 received placebo along with a 52-week corticosteroid taper.
The study met its primary endpoint, with 28% of patients in the sarilumab group achieving sustained remission at 52 weeks vs. 10% in the placebo group (P = .0193), according to the company. The researchers defined sustained remission as remission by week 12 followed by the absence of flare, C-reactive protein normalization from weeks 12 to 52, and adherence to the corticosteroid taper from weeks 12 to 52.
Meanwhile, a sensitivity analysis removing acute phase reactants demonstrated sustained significance (proportion difference = 18%; 95% CI, 3.1% to 32.6%) for the primary outcome.
The median cumulative corticosteroid dose was 777 mg for patients receiving sarilumab, compared with 2,044 mg for those in the placebo group.
Common adverse reactions that occurred in 5% or more of patients in the sarilumab group included: neutropenia, in 15% of patients; leukopenia, 7%; constipation, in 7%; rash pruritic, in 5%; myalgia, in 7%; fatigue, in 5%; and injection site pruritus, in 5%. Serious neutropenia occurred in 3% of patients who received sarilumab vs. 0% in the placebo group.
In both cases of serious neutropenia, the patients demonstrated a neutrophil count of less than 500 per mm3 without any infections. Neutropenia resolved after permanent discontinuation of sarilumab.
The most common adverse events resulting in permanent discontinuation were neutropenia, in three patients, and infection, in three separate patients, including COVID-19, intervertebral discitis and pneumonia.
The FDA had previously approved sarilumab for moderate to severe active rheumatoid arthritis in adults with an inadequate response, or intolerance to, one or more disease-modifying antirheumatic drugs.
“Until now, people living with polymyalgia rheumatica have had limited treatment options for this serious rheumatic condition which causes significant pain and discomfort,” Bill Sibold, executive vice president and head of specialty care at Sanofi, said in the release. “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.”
Read more about
Click Here to Manage Email Alerts