FDA Approves Brepocitinib, First Oral Drug for Dermatomyositis in Adults

The FDA approved brepocitinib (Lisraya) tablets for the treatment of dermatomyositis in adults, making it the first oral drug approved for the condition. The once daily drug, developed by Priovant Therapeutics Inc, received orphan drug and priority review designations and is dosed at 30 mg daily.¹

"For too long, patients with dermatomyositis have faced a significant unmet need for effective treatments," Nikolay Nikolov, MD, director of the Office of Immunology and Inflammation in the FDA's Center for Drug Evaluation and Research, said in a statement.¹

A Rare Disease With Few Treatment Options

Dermatomyositis is a rare, acquired immune-mediated inflammatory myopathy in which the immune system attacks muscle and skin, producing progressive muscle weakness alongside a distinctive rash.^1,2

Incidence is estimated at approximately 9.63 cases per 1,000,000 people annually, with onset most common between ages 40 and 50 years and a higher prevalence among women. Roughly 21% of cases are amyopathic, meaning skin findings appear without muscle weakness.²

Muscle involvement typically affects the trunk and proximal muscles—hips, thighs, shoulders, upper arms, and neck—causing difficulty climbing stairs, rising from a seated position, or lifting objects and, in severe cases, difficulty swallowing or speaking. The characteristic skin findings include a heliotrope rash on the upper eyelids and Gottron papules over the finger joints, along with photosensitive rashes that can be harder to detect on darker skin tones.^2,3

Until now, first-line treatment has centered on high-dose corticosteroids, typically continued for 9 to 12 months, with immunosuppressants such as azathioprine or methotrexate added to limit steroid exposure. Patients with disease resistant to these approaches have been treated with rituximab, intravenous immune globulin (IVIG), or cyclophosphamide.²

Chronic corticosteroid use carries its own risks, including infection, diabetes, cardiovascular disease, osteoporosis, and glucocorticoid myopathy, which is part of what makes an oral, steroid-sparing option clinically meaningful for this population.

VALOR Trial Data Supporting Approval

The approval was based on the Study to Investigate the Efficacy and Safety of Brepocitinib in Adults with Dermatomyositis (VALOR; NCT05437263), a phase 3, multicenter, double-blind, randomized, placebo-controlled trial conducted at 90 sites in 20 countries over 52 weeks.⁴

Investigators enrolled 241 adults with treatment-resistant dermatomyositis—defined as an inadequate response to at least one prior therapy, including systemic glucocorticoids, conventional disease-modifying antirheumatic drugs, or IVIG—and randomly assigned them 1:1:1 to brepocitinib 30 mg, brepocitinib 15 mg, or placebo. The mean patient age was 50.6 years, and 77.6% of participants were women.⁴

At week 52, the mean Total Improvement Score (TIS), a validated composite measure of myositis activity, was 46.5 in the brepocitinib 30-mg group, 37.5 in the 15-mg group, and 31.2 in the placebo group. The least-squares mean difference between the 30-mg dose and placebo was 15.3 points (95% CI, 6.7-24.0; P < .001). The 15-mg dose did not meet the prespecified threshold for statistical significance against placebo, and the FDA approved only the 30-mg dose.⁴

Brepocitinib 30 mg was superior to placebo across all 9 key secondary end points, with clinical benefit observed as early as week 4. By week 52, 68% of patients on the 30-mg dose had a TIS of 40 or higher, indicating moderate improvement, compared with 44% on placebo.⁴

Skin disease activity, measured by the Cutaneous Dermatomyositis Disease Area and Severity Index–Activity score, improved by a mean of 11.7 points from baseline in the 30-mg group versus 7.0 points with placebo (P < .001). Patients on the 30-mg dose also reported greater gains in physical function, with a mean change in Health Assessment Questionnaire–Disability Index score of −0.337 versus −0.042 for placebo.⁴

By weeks 48 through 52, 62% of patients on brepocitinib 30 mg who were taking glucocorticoids at baseline had tapered to 2.5 mg or less of a prednisone equivalent per day, and 42% had tapered to 0 mg, compared with 34% and 23%, respectively, on placebo. More than half of the 30-mg group (54%) achieved a TIS of 40 or higher with minimal-to-no glucocorticoid use, versus 27% on placebo (P < .001).⁴

Safety Profile and Boxed Warnings

Lisraya carries boxed warnings for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events, and thrombosis, consistent with warnings applied to other JAK inhibitors.¹

In the VALOR trial, the overall incidence of adverse events was similar across groups—90% with brepocitinib 30 mg, 86% with the 15-mg dose, and 91% with placebo—but serious infections were more frequent with the 30-mg dose, occurring in 10% of patients compared with 2% on the 15-mg dose and 1% on placebo. The most common adverse events across groups, each occurring in at least 5% of any group, included upper respiratory tract infection, COVID-19, urinary tract infection, nausea, diarrhea, and headache.^1,4

Discontinuation because of adverse events occurred in 6% of patients on brepocitinib 30 mg compared with 11% on placebo, and no deaths were reported during the trial.^1,4

Treatment discontinuation overall was more than twice as frequent in the placebo group as in the 30-mg group (25% versus 11%), and rescue medication was used in 30% of placebo patients compared with 15% of those on the 30-mg dose. Cancers and thromboembolic events in the trial occurred only in the placebo group.⁴

What It Means for Pharmacists

Lisraya's approval gives pharmacists a new oral, steroid-sparing option to discuss with prescribers managing patients whose dermatomyositis has not responded adequately to glucocorticoids, conventional disease-modifying antirheumatic drugs, or IVIG.

Given the boxed warnings shared with other JAK inhibitors, pharmacists should be positioned to counsel patients on signs of infection, monitor for interactions and contraindications relevant to cardiovascular and thromboembolic risk, and support adherence to any required baseline or ongoing laboratory monitoring. The glucocorticoid-tapering data from VALOR also make brepocitinib relevant to medication therapy management conversations aimed at reducing long-term steroid exposure in this population.

REFERENCES

1. FDA approves first oral drug indicated to treat dermatomyositis in adults. News release. U.S. Food and Drug Administration. 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. Idiopathic inflammatory myopathies (dermatomyositis, polymyositis). StatPearls. National Center for Biotechnology Information. Accessed August 27, 2026. https://www.ncbi.nlm.nih.gov/books/NBK558917/

3. Dermatomyositis - symptoms and causes. Mayo Clinic. Accessed August 27, 2026. https://www.mayoclinic.org/diseases-conditions/dermatomyositis/symptoms-causes/syc-20353188

4. Lundberg IE. A Phase 3 Trial of Brepocitinib in Dermatomyositis. N Engl J Med. 2026;394(19):1953-1955. doi:10.1056/NEJMe2604009