FDA Approves Oral Drug for Dermatomyositis in Adults
The U.S. Food and Drug Administration has approved brepocitinib (Lisraya) tablets for the treatment of dermatomyositis in adults.
Brepocitinib is a a once-daily oral Janus kinase inhibitor that blocks JAK pathways to reduce the chronic inflammation, progressive muscle weakness, and distinctive skin rashes that characterize dermatomyositis, a rare autoimmune disease.
The agency evaluated the efficacy and safety of brepocitinib in a phase 3 randomized, double-blind, multicenter, placebo-controlled study that included 241 adults with dermatomyositis. Participants were randomized to receive brepocitinib 30 mg once daily, brepocitinib 15 mg once daily, or placebo. The Total Improvement Score (TIS), a standardized scoring tool, tracked changes across muscle strength; physical function; skin and other disease activity; muscle enzymes; and both physician and patient assessments of overall health, at week 52 of the trial to assess improvements.
Participants treated with brepocitinib 30 mg had a higher average TIS score, indicating greater clinical response and disease control, at week 52 compared to those who received a placebo. They also showed improvements in physical function and skin disease activity and were more likely to reduce corticosteroid use by week 48.
Some of the most common adverse reactions to brepocitinib were upper respiratory tract infection, headache, fatigue, urinary tract infection, and nausea. Discontinuation due to adverse reactions occurred in 6% of participants treated with brepocitinib 30 mg, compared with 11% of those given a placebo. Brepocitinib carries a boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular, and thrombosis.
Reference: FDA approves first oral drug indicated to treat dermatomyositis in adults. News release. US Food and Drug Administration; August 27, 2026. Accessed August 27, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-drug-indicated-treat-dermatomyositis-adults?utm_medium=email&utm_source=govdelivery
