The U.S. Food and Drug Administration (FDA) has approved Priovant Therapeutics’ LISRAYA (brepocitinib) 30 mg for the treatment of adults with dermatomyositis (DM), a rare autoimmune disease that can cause muscle weakness and painful skin problems.
LISRAYA is a once-daily oral medicine and is the first targeted therapy approved specifically for dermatomyositis, according to Priovant. The company said the treatment can improve several areas of the disease, including skin symptoms, muscle strength, physical function and overall disease activity, while also helping reduce the need for long-term steroid treatment.
Dermatomyositis can have a major effect on patients’ daily lives. The disease is marked by progressive muscle weakness and skin lesions that can be painful, itchy and sensitive to light and touch. Many patients have traditionally been treated with steroids and other non-targeted immune therapies.
The FDA approval is based on results from the Phase 3 VALOR trial, described by Priovant as the largest clinical trial conducted in dermatomyositis. The study evaluated LISRAYA in patients with different levels of disease activity and across a range of background treatments.
In the trial, improvements in the primary measure, the Myositis Total Improvement Score, were seen as early as four weeks after treatment. Benefits increased over time and were maintained through the 52-week study.
By the end of the study, 55% of patients receiving LISRAYA achieved moderate or greater improvement on the Total Improvement Score while also reaching minimal or no steroid use. This compared with 30% of patients receiving placebo.
LISRAYA also showed a steroid-sparing effect among patients taking at least 7.5 mg per day of prednisone or an equivalent corticosteroid at the start of the study. About 62% reduced their steroid dose to 2.5 mg per day or less after 52 weeks, compared with 38% of patients receiving placebo. In addition, 45% of LISRAYA-treated patients stopped corticosteroids completely, compared with 29% in the placebo group.
The treatment also improved measures of skin disease and muscle strength. Patients taking LISRAYA reported greater improvements in their overall assessment of disease activity compared with those receiving placebo. The trial also found improvements in everyday activities such as getting dressed, climbing stairs and running errands.
Priovant said LISRAYA can be prescribed immediately in the United States. The medicine will be available through a limited network of specialty pharmacies, while the company’s patient support program will provide assistance with insurance coverage and financial support for eligible patients.
The most common side effects reported in the VALOR trial included upper respiratory tract infections, headache, fatigue, urinary tract infections, nausea, bronchitis, joint pain, diarrhea, back pain, falls, influenza and acne.
The medicine also carries important safety warnings related to serious infections, malignancies, major cardiovascular events and blood clots. Patients may require monitoring for infections and certain laboratory abnormalities during treatment.
The FDA had previously granted LISRAYA Priority Review and Orphan Drug Designation. The primary results from the VALOR trial were published in the New England Journal of Medicine in March 2026, while additional skin-related results were published in JAMA Dermatology in August 2026.
With the approval, LISRAYA becomes a new targeted treatment option for adults with dermatomyositis and provides an oral therapy aimed at addressing multiple aspects of the disease while potentially reducing dependence on corticosteroids.