The US FDA has approved Lisraya (brepocitinib) 30 mg for the treatment of adults with dermatomyositis, making it the first targeted therapy approved for the rare systemic autoimmune disease. Roivant Sciences (Nasdaq: ROIV) and its subsidiary Priovant Therapeutics received the approval, which carries no restrictions on disease activity level, clinical presentation, or prior treatment experience.
Lisraya is administered as a once-daily oral pill. The label includes a boxed warning covering serious infections, mortality, malignancy, major adverse cardiovascular events, and thrombosis — consistent with the broader JAK inhibitor class. It is not recommended in combination with other JAK inhibitors, TYK2 inhibitors, or biologic disease-modifying antirheumatic drugs (DMARDs), nor in patients with severe hepatic or renal impairment. The FDA had previously granted Priority Review and Orphan Drug Designation for the application, as reported following NDA acceptance in March 2026.
The approval was supported by the Phase III VALOR trial, described by the company as the largest dermatomyositis clinical trial ever conducted. The primary endpoint was the myositis Total Improvement Score (TIS), a composite measure across multiple disease domains. At 52 weeks, 55% of brepocitinib-treated patients achieved moderate or better TIS improvement alongside minimal or no steroid use, compared with 30% on placebo. Among patients on corticosteroids at baseline, 62% tapered to minimal or no steroid use by week 52 versus 38% on placebo; 45% discontinued corticosteroids entirely compared with 29% on placebo. Primary results were published in the New England Journal of Medicine in March 2026, with skin-specific secondary endpoints published in JAMA Dermatology in August 2026.