The VALOR phase 3 trial shows that brepocitinib 30 mg daily — a selective TYK2–JAK1 inhibitor — significantly improves composite disease activity, skin involvement, and muscle strength while enabling steroid discontinuation in adults with treatment-refractory dermatomyositis, marking the first successful phase 3 RCT of an oral targeted agent in this disease.
A contemporary NEJM review of idiopathic inflammatory myopathies — the five-subtype framework (IBM, IMNM, antisynthetase syndrome, overlap, dermatomyositis), the autoantibody revolution that now anchors classification, and the shift toward subtype-specific therapy with biologics, JAK/TYK2 inhibitors, and CAR-T.
CD19 CAR-T cellular therapy delivers drug-free remission in 80–85% of refractory SLE patients in early-phase trials, with similar signals in IIM and SSc — the first credible attempt at true immunological reset in autoimmune rheumatic disease.