FDA approves Lisraya, first oral treatment for adult dermatomyositis

Dermatomyositis can cause progressive muscle weakness, disabling skin disease, and systemic complications, yet treatment has long relied largely on corticosteroids and immunosuppressive therapies approved for other conditions. FDA approved Lisraya (brepocitinib), the first oral therapy specifically indicated for adults with dermatomyositis.

Lisraya is a once-daily selective TYK2/JAK1 inhibitor that targets inflammatory signaling involved in dermatomyositis. The oral option may provide an alternative to intravenous immune globulin and could help some patients reduce corticosteroid exposure, although treatment will remain specialist-directed and may continue alongside other therapies.

Approval was based on a 52-week phase 3 trial of 241 adults whose dermatomyositis had not responded adequately to previous treatment. Participants continued standard therapies while receiving brepocitinib or placebo and tapering glucocorticoids.

The primary endpoint was the Total Improvement Score, a 100-point composite measuring muscle strength, physical function, skin and other disease activity, muscle enzymes, and overall health assessments. At week 52, the mean score was 46.5 with the approved 30 mg dose versus 31.2 with placebo, a 15.3-point difference. Improvements were evident by week four and extended to skin disease, functional disability, and corticosteroid tapering. The 15 mg dose did not significantly improve the primary endpoint.

The approval adds an important option but also introduces JAK inhibitor safety considerations. Serious infections occurred in 10% of patients receiving Lisraya 30 mg versus 1% with placebo. The drug carries a boxed warning for serious infections, mortality, malignancies, major cardiovascular events, and thrombosis, making individual risk assessment and ongoing monitoring central to treatment decisions.

“Dermatomyositis affects nearly every aspect of a patient’s life, causing physical disability, disfiguring skin disease, pain, itch, and a profound loss of independence and sense of self,” said Ruth Ann Vleugels, MD, professor of dermatology at Harvard Medical School. “For many decades, the treatment of dermatomyositis has relied on chronic steroids, non-specific immunomodulators, and intravenous immunoglobulin—therapies not targeted to the underlying disease pathobiology. The approval of Lisraya marks a turning point for patients living with dermatomyositis.”

Source: FDA. (2026 Aug 27). FDA approves first oral drug indicated to treat dermatomyositis in adults