Difelikefalin for IV use
Trade Name: Korsuva™
Company: Vifor Pharma, Cara Therapeutics
Approval Dates/Comments: In August 2021, the US FDA approved this first-in-class, selective peripheral kappa opioid receptor agonist for the treatment of moderate-to-severe pruritus associated with chronic kidney disease (CKD-aP) in adults undergoing hemodialysis. An oral formulation of difelikefalin is also in development for pruritus associated with atopic dermatitis.
Topical MEK inhibitor
Trade Name: NFX-179
Company: NFlextion Therapeutics
Approval Dates/Comments: In August 2021, the FDA granted Orphan Drug designation for NFX-179 for the treatment of cutaneous neurofibromatosis type 1 (NF1). NFX-179 is a topical, first-in-class, “soft” (metabolically labile) mitogen-activated protein kinase kinase (MEK) inhibitor that is currently being investigated in Phase 2 clinical trials for cutaneous NF1.
Maralixibat oral solution
Trade Name: Livmarli™
Company: Mirum Pharmaceuticals
Approval Dates/Comments: FDA approval was granted in September 2021 for maralixibat (a minimally absorbed ileal bile acid transporter inhibitor) as the first and only approved medication to treat cholestatic pruritus in patients with Alagille syndrome aged ≥1 year.
Ruxolitinib cream 1.5%
Trade Name: Opzelura™
Approval Dates/Comments: The FDA approved this first-in-class, selective, topical Janus kinase (JAK) 1/JAK2 inhibitor in September 2021 for the short-term and noncontinuous chronic treatment of mild to moderate atopic dermatitis (AD) in nonimmunocompromised patients ≥12 years of age whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable. The approval was based on data from TRuE-AD1 and TRuE-AD2 trials, which demonstrated significantly clearer skin and itch reduction in patients using ruxolitinib 1.5% cream. Additionally, significantly more patients treated with ruxolitinib cream achieved Investigator’s Global Assessment (IGA) Treatment Success (IGA-TS; primary endpoint) at week 8 (defined as an IGA score of 0 [clear] or 1 [almost clear] with at least a 2-point improvement from baseline): 53.8% in TRuE-AD1 and 51.3% in TRuE-AD2, compared with nonmedicated treatment (15.1% in TRuE-AD1, 7.6% in TRuE-AD2; P<0.0001). Opzelura™ carries a class-wide boxed warning for serious infections, mortality, cancer, major adverse cardiovascular events, and thrombosis, which is based on data from the oral JAK inhibitor tofacitinib in rheumatoid arthritis. This drug is also being developed for vitiligo.
Trade Name: Jakafi®
Approval Dates/Comments: In September 2021, the FDA approved oral ruxolitinib for chronic graft-versus-host disease (GVHD) after failure of 1 or 2 lines of systemic therapy in patients ≥12 years of age. This marks the fourth FDA-approved indication for Jakafi®.
Trade Name: Tavneos™
Approval Dates/Comments: The FDA approved avacopan in October 2021 as an addon treatment to standard therapy including glucocorticoids for adults with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis. This orally administered selective complement 5a receptor inhibitor is indicated for granulomatosis with polyangiitis and microscopic polyangiitis, which are the 2 main forms of ANCA vasculitis.