- Design
- Prespecified secondary analysis of a phase 3, double-blind, placebo-controlled RCT, 52 weeks
- Population
- 241 adults with active dermatomyositis, 90 sites in 20 countries
- Primary outcome
- Change in CDASI-A and CDASI-A response
- Effect
- CDASI-A difference -3.0 (-4.6 to -1.4); response 33.3% vs 17.7%
VALOR randomised 241 adults with active skin and muscle dermatomyositis at 90 sites in 20 countries to brepocitinib 30 mg, 15 mg or placebo once daily for 52 weeks. This prespecified secondary analysis focused on the skin.
With 30 mg, cutaneous activity (CDASI-A) fell more than with placebo from week 4 (-6.4 vs -3.5; difference -3.0, 95% CI -4.6 to -1.4). A clinically meaningful skin response occurred in 33.3% versus 17.7%, itch remission in 38.3% versus 19.0%, and skin-related quality of life improved (Skindex-16 difference -11.9). In those with moderate to severe skin disease, 45.7% versus 21.8% reached clear or almost clear skin at 52 weeks. Safety resembled approved JAK and TYK2 inhibitors.
Cutaneous dermatomyositis is often the most refractory part of the disease and the most itchy. This is the first phase 3 evidence that an oral TYK2/JAK1 inhibitor can control it durably. Brepocitinib is not yet licensed.
- Assess skin activity and itch separately from muscle disease at every dermatomyositis review
- Expect JAK/TYK2 inhibition to act within four weeks on the skin
- Screen for tuberculosis, hepatitis B and herpes zoster risk before any JAK-class drug
- Continue malignancy screening in adult dermatomyositis, whatever the treatment
Why it matters
Refractory skin disease in dermatomyositis has had no drug with phase 3 skin data until now.
Don't overread it
These are secondary and exploratory end points of the trial; the drug has no marketing approval.
The statistics, in plain English
A 15-point difference in response (95% CI 1.7 to 28.6) is significant but imprecise. Exploratory outcomes such as itch remission were not the trial's main tests, so they carry less weight.
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