Brepocitinib Achieves Rapid and Durable Skin Disease Control in Dermatomyositis: JAMA
USA: Researchers have found in a secondary analysis of a randomized trial that once-daily brepocitinib 30 mg provided rapid, durable, and remission-level improvement in cutaneous manifestations of dermatomyositis, with an acceptable safety profile. These findings support brepocitinib as a targeted treatment option for controlling skin disease in dermatomyositis.
The study, published in JAMA Dermatology, was led by Aaron R. Mangold, Department of Dermatology, Mayo Clinic, Scottsdale, Arizona, and colleagues. Investigators assessed the effects of brepocitinib on skin disease activity, itching, and skin-related quality of life (QOL) in adults with dermatomyositis over 52 weeks.
Dermatomyositis is an inflammatory disease that can cause substantial skin and muscle involvement. Brepocitinib is an oral, selective TYK2 and JAK1 inhibitor that has demonstrated broad efficacy in patients with the condition. The current analysis used data from the phase 3, double-blind, placebo-controlled VALOR trial, conducted between October 2022 and July 2025 across 90 sites in 20 countries.
The analysis included 241 adults with active skin and muscle disease, with a mean age of 50.6 years; approximately 78% were women. Participants received once-daily brepocitinib 30 mg, brepocitinib 15 mg, or placebo. Skin disease activity was assessed using the Cutaneous Dermatomyositis Disease Area and Severity Index–Activity (CDASI-A).
The trial revealed the following findings:
- By week 4, brepocitinib 30 mg produced a greater reduction in CDASI-A scores than placebo (−6.4 vs −3.5; between-group difference, −3.0).
- A clinically meaningful CDASI-A response was achieved by 33.3% of patients receiving brepocitinib 30 mg versus 17.7% with placebo.
- Itch remission was achieved by 38.3% of patients receiving brepocitinib compared with 19.0% receiving placebo.
- Skin-related quality of life improved more with brepocitinib, with Skindex-16 scores decreasing by 12.9 points versus 0.9 points with placebo (between-group difference, −11.9).
- Improvements in skin disease activity, itching, and skin-related quality of life were sustained from week 4 through week 52.
- Among patients with moderate-to-severe skin disease at baseline, 45.7% of those receiving brepocitinib achieved clear or almost clear skin at week 52, compared with 21.8% receiving placebo.
- Functional skin remission at week 52 was achieved by 43.5% of patients receiving brepocitinib versus 20.8% receiving placebo.
- Brepocitinib demonstrated a safety profile consistent with approved JAK and TYK2 inhibitors.
One limitation was that background disease-modifying antirheumatic drug therapy had to remain unchanged during the blinded treatment period, which may not fully reflect routine clinical practice where treatment may be reduced after disease control.
The researchers concluded that brepocitinib 30 mg can provide rapid, sustained and remission-level control of cutaneous dermatomyositis, including in patients with substantial baseline skin involvement. Ongoing open-label extension data may provide further insight into its use alongside more flexible background treatment.
Reference:
Mangold AR, Haemel A, Shahriari N, et al. Skin-Specific Outcomes of Brepocitinib in Patients With Dermatomyositis: Secondary Analysis of a Phase 3 Randomized Clinical Trial. JAMA Dermatol. Published online August 26, 2026. doi:10.1001/jamadermatol.2026.3199
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