FDA approves brepocitinib, the first oral drug for dermatomyositis in adults
The U.S. Food and Drug Administration approved LISRAYA (brepocitinib) on August 27, 2026, as the first oral treatment for adults with dermatomyositis. According to Priovant Therapeutics, the once-daily 30 mg drug works as a TYK2/JAK1 inhibitor, targeting specific pathways involved in the autoimmune disease.
The approval of LISRAYA (brepocitinib) marks the first targeted oral therapy authorized by the U.S. Food and Drug Administration specifically for adults with dermatomyositis, a rare autoimmune disease characterized by muscle weakness and a painful skin rash, according to the Muscular Dystrophy Association (MDA). The FDA granted approval on August 27, 2026, following the agency’s acceptance of Priovant Therapeutics’ New Drug Application with Priority Review in March 2026. The Prescription Drug User Fee Act target action date was set for the third quarter of 2026, and Priovant anticipates launching the drug in the United States by the end of September 2026.
Patients receiving brepocitinib 30 mg achieved a 15.3-point greater improvement in mean Total Improvement Score compared to placebo (46.5 vs. 31.2; P<0.001), according to published data and company summaries.
Brepocitinib, marketed under the brand name LISRAYA, is a once-daily 30 mg oral therapy that acts as a TYK2/JAK1 inhibitor, targeting specific signaling pathways involved in autoimmune and inflammatory diseases, according to Priovant Therapeutics and clinical trial data. This mechanism distinguishes it from traditional steroid-based immunosuppressants commonly used in dermatomyositis treatment, which often have incomplete efficacy and significant side effects.
The FDA’s decision was based on results from the pivotal Phase 3 VALOR trial, which enrolled 241 patients at 90 sites worldwide. The primary endpoint was the mean Total Improvement Score (TIS) at week 52, a composite index measuring disease activity across multiple domains. Patients receiving brepocitinib 30 mg achieved a 15.3-point greater improvement in mean TIS compared to placebo (46.5 vs. 31.2; P<0.001), according to published data and company summaries. The 15 mg dose showed a smaller, statistically non-definitive difference versus placebo. Institutional commentary on the VALOR trial noted that nearly 70% of patients on the 30 mg dose achieved moderate or greater improvement, with almost half reaching major improvement in disease activity.
Additional clinical outcomes from the VALOR study demonstrated that brepocitinib 30 mg provided significant benefits in skin disease severity, functional disability, and glucocorticoid tapering. Among patients with moderate-to-severe skin involvement, 44% on the 30 mg dose achieved cutaneous clinical remission by week 52, compared with 21% on placebo, according to Medscape reporting. The drug also showed improvements in the Cutaneous Dermatomyositis Area and Severity Index, motor strength, and the Health Assessment Questionnaire Disability Index. The ability to reduce reliance on systemic glucocorticoids was highlighted, with 62% of patients on brepocitinib 30 mg achieving a steroid dose of 2.5 mg per day or less by the end of the study, compared to 34% on placebo. Additionally, 42% of patients on brepocitinib discontinued steroids completely by week 52, versus 23% in the placebo group.
Safety data from the VALOR trial indicated a higher incidence of serious infections in the brepocitinib 30 mg group compared to placebo (10% vs. 1%), although no deaths occurred during the study period. The TYK2/JAK1 inhibitor mechanism carries class-specific risks, including infection, which was emphasized in expert commentaries and trial summaries. Despite the increased risk of serious infections, the overall safety profile was described as manageable in patients with refractory dermatomyositis.
Dermatomyositis has historically been treated with systemic glucocorticoids and broad immunosuppressants, which often provide incomplete disease control and carry significant side effects, according to medical literature and prior treatment guidelines. The MDA welcomed the FDA approval of LISRAYA as a major advance, calling it the first targeted therapy approved specifically for adults living with dermatomyositis. Roivant Sciences, through its affiliate Priovant Therapeutics, is the developer and sponsor of brepocitinib for this indication. Media coverage on August 27, 2026, including reports by STAT and Medscape, described the approval as a notable milestone in rare autoimmune disease therapeutics.
The VALOR trial’s positive results and the FDA’s approval signal a potential shift in the treatment landscape for dermatomyositis, offering an oral, targeted option where none previously existed. Further post-marketing surveillance and real-world data will be needed to monitor long-term safety and effectiveness as brepocitinib becomes available to patients.
