FDA Approvals, Highlights, and Summaries: Rheumatology

February 17, 2022

Saphnelo (anifrolumab)

Anifrolumab is an IgG1 monoclonal antibody that binds to subunit 1 of the type I interferon (IFN) receptor. Binding inhibits type 1 IFN signaling, thereby blocking biologic activity of type 1 IFNs (eg, plasma cell differentiation) and normalizes peripheral T-cell subsets. Anifrolumab is indicated for treatment of moderate-to-severe systemic lupus erythematosus.

Approval was based in part on combined data from the TULIP-2 phase 3 trial and the MUSE phase II trial. Patients in the TULIP-2 trial who received anifrolumab had a higher British Isles Lupus Assessment Group (BILAG)–based Composite Lupus Assessment (BICLA) response as compared to placebo (47.8% vs 31.5%; P=0.001) N Engl J Med. 2020;382(3):211-221

The MUSE trial compared 2 doses of anifrolumab (300 mg or 1000 mg) with placebo. Anifrolumab substantially reduced disease activity when compared to placebo across primary and secondary endpoints in patients with moderate-to-severe SLE. Rheumatology. 2021 Dec 1;60(12):5854-5862

Tavneos (avacopan)

Avacopan is indicated as adjunctive treatment of severe active antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis (granulomatosis with polyangiitis and microscopic polyangiitis) in combination with glucocorticoids. Avacopan is a complement 5a receptor (C5aR) antagonist that inhibits the interaction between C5aR and the anaphylatoxin C5a.

Results from the phase 3 ADVOCATE trial compared avacopan (n = 166) with oral prednisone (n = 165) for treatment of ANCA-associated vasculitis. All patients received cyclophosphamide or rituximab. The mean Birmingham Vasculitis Activity Score (BVAS) at baseline was 16 in both groups. Remission at Week 26 was observed in 72.3% receiving avacopan and in 70.1% receiving prednisone (P< 0.001 for noninferiority; P=0.24 for superiority). Sustained remission at Week 52 was observed in 65.7% receiving avacopan and 54.9% receiving prednisone (P< 0.001 for noninferiority; P=0.007 for superiority). N Engl J Med. 2021 Feb 18;384(7):599-609

Other rheumatology approvals

Rinvoq (upadacitinib) - New indication for active psoriatic arthritis (PsA) in adults who have had an inadequate response or intolerance to 1 or more TNF blockers.

Xeljanz (tofacitinib) - New indication for active ankylosing spondylitis.

Octagam (immune globulin IV) - First IVIG approved for treatment of dermatomyositis in adults.

 

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