Lupus Drug Is Approved Despite Previous Clinical Trial Setback | Rheumatology | JAMA | JAMA Network
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News From the Food and Drug Administration
September 21, 2021

Lupus Drug Is Approved Despite Previous Clinical Trial Setback

JAMA. 2021;326(11):1000. doi:10.1001/jama.2021.14964

The FDA has approved anifrolumab-fnia for patients with systemic lupus erythematosus (SLE) even though the initial phase 3 trial results showed the drug didn’t outperform placebo in meeting its primary end point.

Corticosteroids, nonsteroidal anti-inflammatory medications, and the antimalarial drug hydroxychloroquine, all of which are standard treatments for SLE, can cause organ damage and other adverse events. Monoclonal antibodies like anifrolumab-fnia can cause adverse events, too, but they also may reduce the need for corticosteroids. Anifrolumab-fnia improves symptoms by inhibiting type I interferon signaling, which is increased in people with SLE and is associated with more severe disease. Its predecessor, belimumab, blocks B lymphocyte stimulator, which reduces autoreactive B cells involved in SLE pathogenesis.

In the initial phase 3 clinical trial, 36% of patients who received anifrolumab-fnia compared with 40% in the placebo group achieved an SLE responder index 4 (SRI-4) response—a composite measure of improvement and the trial’s end point. However, patients taking the drug reduced their corticosteroid use and fared better than those in the placebo group on another measure of improvement, the British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA).

The second phase 3 trial used BICLA response as its primary end point. Results showed that 47.8% of the 180 patients taking anifrolumab-fnia improved as measured by BICLA response compared with 31.5% of the 182 patients who received placebo. Patients in the treatment group also reduced their corticosteroid use and had less severe skin disease. About 7% developed herpes zoster and 12% developed bronchitis while taking the drug. Other reported adverse events included nasopharyngitis and upper respiratory tract infection.

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