FDA Approves LISRAYA as First Targeted Oral Therapy for Adults with Dermatomyositis

The U.S. Food and Drug Administration (FDA) has approved LISRAYA™ (brepocitinib) 30 mg once daily for adults with dermatomyositis (DM), introducing the first FDA-approved oral therapy for the rare systemic autoimmune disease. The approval is supported by the Phase 3 VALOR trial, in which the 30 mg dose significantly improved the Total Improvement Score (TIS) and all nine key secondary endpoints versus placebo.

A targeted approach to dermatomyositis

Dermatomyositis causes progressive muscle weakness and inflammatory skin disease that can produce painful, itchy and disfiguring lesions. The condition can substantially impair physical function and independence, while treatment has historically relied on corticosteroids, immunosuppressants and other non-specific therapies.

LISRAYA is a first-in-class oral TYK2/JAK1 inhibitor that blocks cytokine signaling implicated in dermatomyositis. The FDA-approved 30 mg regimen is indicated for adults with DM without restrictions based on disease activity, clinical presentation or previous treatment experience. It can be used as an alternative or alongside non-targeted DM therapies, although the prescribing information advises against combining it with other JAK inhibitors, TYK2 inhibitors or biologic DMARDs.

VALOR established the 30 mg dose

The pivotal Phase 3 VALOR trial (NCT05437263) randomized 241 adults with dermatomyositis to once daily brepocitinib 30 mg, brepocitinib 15 mg or placebo for 52 weeks. The primary endpoint was TIS, a validated composite measure of improvement across multiple domains of myositis.

At Week 52, mean TIS was 46.5 with 30 mg brepocitinib versus 31.2 with placebo, an adjusted difference of 15.3 points (95% CI, 6.7-24.0; P<0.001). The 15 mg dose produced a mean TIS of 37.5, but its 6.3-point difference versus placebo was not statistically significant. Importantly, 30 mg was superior to placebo across all nine key secondary endpoints, while 15 mg did not establish the same efficacy profile.

Benefits with 30 mg emerged as early as Week 4 and extended across skin disease, muscle strength, physical function and corticosteroid tapering. The VALOR findings therefore supported the specific 30 mg once-daily regimen approved by the FDA.

The steroid-sparing effect was particularly notable. Among patients receiving at least 7.5 mg/day of prednisone-equivalent corticosteroids at baseline, 62% of those receiving LISRAYA reduced steroid use to 2.5 mg/day or less by Week 52, compared with 38% on placebo. Complete corticosteroid discontinuation occurred in 45% and 29% of patients, respectively.

Safety remains an important consideration

Serious infections occurred more frequently with 30 mg brepocitinib than placebo in VALOR, at 10% versus 1%, although no deaths occurred during the trial. Common adverse reactions included upper respiratory tract infection, headache, fatigue, urinary tract infection, nausea, bronchitis, arthralgia, diarrhea, back pain, falls, influenza and acne.

LISRAYA carries a boxed warning for serious infections, increased all-cause mortality, malignancy, major adverse cardiovascular events (MACE), and thrombosis.

Regulatory milestone and U.S. launch

The FDA granted LISRAYA Orphan Drug and Priority Review designations before approval. Priovant is making the drug available immediately in the U.S. through a limited specialty pharmacy network, with patient access and financial assistance available through its My Compass Support program. Eligible patients may pay as little as $0 per month.

The primary VALOR findings appeared in the New England Journal of Medicine in the May 14, 2026 issue, following online publication in March, while additional skin-specific analyses were published in JAMA Dermatology in August.

The approval establishes brepocitinib as a new targeted treatment option for dermatomyositis, with clinical evidence spanning disease activity, cutaneous and muscle manifestations, functional outcomes and corticosteroid reduction.

Reference

FDA Approves First Oral Drug Indicated to Treat Dermatomyositis in Adults | FDA

Priovant Announces FDA Approval of LISRAYA™ (brepocitinib)