FDA Approves LISRAYA (brepocitinib): First Oral Targeted Therapy for Adults with Dermatomyositis

　　On August 27,2026,the U.S.FDA approved LISRAYA(brepocitinib)30 mg,a once-daily oral tablet,for the treatment of dermatomyositis(DM)in adults.It is the first targeted therapy and the first oral agent ever approved for the disease,following prior Orphan Drug and Priority Review designations.

　　DM is a rare systemic autoimmune disease characterized by progressive muscle weakness and distinctive skin rash;severe cases can compromise swallowing and respiration.Historically,management relied on systemic corticosteroids,non-specific immunosuppressants and intravenous immunoglobulin,with no approved agent directed at disease biology.Brepocitinib is a first-in-class oral selective dual TYK2/JAK1 inhibitor that blocks TYK2-and JAK1-mediated signal transduction,suppressing type I and type II interferons as well as IL-6,IL-12 and IL-23.

　　Approval was supported by the pivotal phase 3 VALOR trial(NCT05437263),which randomized 241 adults with refractory DM 1:1:1 to brepocitinib 30 mg,15 mg or placebo for 52 weeks across 90 sites in 20 countries.At Week 52,mean Total Improvement Score(TIS)was 46.5 with the 30 mg dose versus 31.2 with placebo(difference,15.3;95%CI,6.7–24.0;P<0.001),and the 30 mg arm was superior across all nine key secondary endpoints,covering skin disease activity,muscle strength,physical function and glucocorticoid tapering.The 15 mg dose did not separate from placebo.Steroid sparing was clinically meaningful:among patients receiving≥7.5 mg/day prednisone equivalent at baseline,62%on 30 mg tapered to≤2.5 mg/day by Week 52 versus 38%on placebo,and 45%discontinued corticosteroids entirely versus 29%.

　　The most common adverse reactions included upper respiratory tract infection,headache,fatigue,urinary tract infection and nausea.Serious infections occurred in approximately 10%of patients on 30 mg versus 1%on placebo,with no deaths during the trial;discontinuation due to adverse reactions was 6%versus 11%.LISRAYA carries a boxed warning for serious infections,all-cause mortality,malignancy,major adverse cardiovascular events(MACE)and thrombosis.

　　The recommended regimen is 30 mg orally once daily,with or without food,with no restriction based on disease activity,clinical presentation or prior treatment experience.Concomitant use with other JAK inhibitors,TYK2 inhibitors or biologic DMARDs is not recommended.The approval moves DM management away from chronic corticosteroid dependence toward targeted oral therapy.