FDA Approves First Oral Drug for Dermatomyositis

The FDA has approved brepocitinib (Lisraya; Priovant Therapeutics) tablets for the treatment of dermatomyositis in adults, making it the first oral therapy indicated for the rare autoimmune disease. For a patient population that has long depended on high-dose chronic corticosteroids and therapies borrowed from other conditions, the approval gives pharmacists a new, targeted oral option to counsel patients on and to monitor.¹

A First-in-Class Oral Option for a Rare Disease

Dermatomyositis is a rare autoimmune disease in which the immune system attacks the muscles and skin, producing chronic inflammation, progressive muscle weakness, and distinctive skin rashes that are often disfiguring and painful. Brepocitinib is a first-in-class, once-daily oral, selective tyrosine kinase 2-Janus kinase 1 (TYK2-JAK1) inhibitor that blocks cytokine signaling implicated in dermatomyositis; by dampening these immune and inflammatory pathways, it helps reduce the inflammation that damages muscle and skin. The FDA granted the approval to Priovant Therapeutics and gave brepocitinib both Orphan Drug and Priority Review designations.^1,2

What the VALOR Trial Showed

Efficacy and safety were evaluated in the phase 3 VALOR trial (NCT05437263), a randomized, double-blind, multicenter, placebo-controlled study that enrolled 241 adults with dermatomyositis whose disease was resistant to previous therapy. Participants were randomized 1:1:1 to brepocitinib 30 mg once daily (n = 81), brepocitinib 15 mg once daily (n = 81), or placebo (n = 79) for 52 weeks, with standard therapies continued and glucocorticoids tapered. The primary endpoint was the Total Improvement Score (TIS)—a validated composite myositis index ranging from 0 to 100, with higher scores indicating greater improvement—at week 52.^1-3

At week 52, the mean TIS was 46.5 in the brepocitinib 30-mg group, 37.5 in the 15-mg group, and 31.2 with placebo (difference for 30 mg vs placebo, 15.3; 95% CI, 6.7 to 24.0; P < 0.001). The 15-mg dose did not separate significantly from placebo (difference, 6.3; 95% CI, −2.4 to 14.9). Brepocitinib 30 mg was superior to placebo across all 9 key secondary endpoints—including skin disease activity, systemic glucocorticoid tapering, and functional disability—with improvements seen as early as week 4. Patients on the 30-mg dose were also more likely to reduce corticosteroid use.^1,2

Safety Profile and Counseling Considerations

The most common adverse reactions included upper respiratory tract infection, headache, fatigue, urinary tract infection, and nausea. Serious infections were more frequent with brepocitinib 30 mg than placebo (10% vs 1%), though no deaths occurred during the trial. Discontinuation due to adverse reactions occurred in 6% of participants on brepocitinib 30 mg versus 11% on placebo.^1,2

Pharmacists should note that brepocitinib carries a boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events, and thrombosis, a class warning familiar from other JAK inhibitors. That makes infection screening, cardiovascular and malignancy risk assessment, and patient counseling on signs of infection and clotting central to dispensing and follow-up.¹

REFERENCES

1. FDA approves first oral drug indicated to treat dermatomyositis in adults. News release. News release. FDA. Released August 27, 2026. Accessed August 27, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-drug-indicated-treat-dermatomyositis-adults

2. Vleugels RA, Paik JJ, Bauer Ventura I, et al; VALOR Investigators. A phase 3 trial of brepocitinib in dermatomyositis. N Engl J Med. 2026;394(19):1883-1893. doi:10.1056/NEJMoa2503531

3. A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis (VALOR). ClinicalTrials.gov Identifier: NCT05437263. Last Updated July 17, 2026. Accessed August 27, 2026. https://clinicaltrials.gov/study/NCT05437263