FDA Approves First Oral Treatment of Dermatomyositis in Adult Patients

The US Food and Drug Administration (FDA) has approved brepocitinib (Lisraya) tablets for the treatment of dermatomyositis in adults, adding a once-daily oral Janus kinase (JAK/TYK2) inhibitor specifically studied in this population.¹

Dermatomyositis has historically been managed with therapies developed for other conditions, leaving specialists without an agent evaluated specifically in this population.¹ Brepocitinib's approval, granted to Priovant Therapeutics Inc, adds an on-label oral option backed by dedicated phase 3 data.¹ The FDA granted Lisraya Orphan Drug and Priority Review designations ahead of this decision.¹

"For too long, patients with dermatomyositis have faced a significant unmet need for effective treatments, often relying on therapies meant for other diseases," Nikolay Nikolov, MD, director of the Office of Immunology and Inflammation, FDA Center for Drug Evaluation and Research, said in a statement. "Today's approval is a meaningful step forward, giving patients and their healthcare providers an approved oral therapy proven to help manage this rare and debilitating disease."

Brepocitinib Efficacy in Phase 3 Dermatomyositis Trial

The approval rests on a phase 3 randomized, double-blind, multicenter, placebo-controlled trial (NCT05437263) enrolling 241 adults with dermatomyositis.² Investigators randomized participants to brepocitinib 30 mg once daily, brepocitinib 15 mg once daily, or placebo across a 52-week treatment period.¹ Evaluating both doses against placebo allowed investigators to compare dose-dependent efficacy and tolerability within the same trial.¹

The primary endpoint was Total Improvement Score (TIS) at week 52, a composite measure spanning 6 domains.¹ Those domains include muscle strength, physical function, skin and other disease activity, muscle enzymes, and physician and patient global assessments of overall health.¹ Participants treated with brepocitinib 30 mg achieved a higher average TIS at week 52 than those receiving placebo, reflecting greater clinical response and disease control.¹

Brepocitinib inhibits Janus kinase and TYK2 signaling, pathways central to the immune and inflammatory activity underlying dermatomyositis.¹ By blocking this signaling, the therapy is designed to reduce inflammation driving muscle and skin involvement.¹ The FDA based its approval on this mechanism together with the trial's clinical results.¹

Brepocitinib Safety Profile and Secondary Outcomes in Dermatomyositis

Participants receiving brepocitinib 30 mg also showed improvements in physical function and skin disease activity relative to placebo through week 52.¹ They were more likely to reduce corticosteroid use by week 48 than participants receiving placebo, a secondary marker of disease control beyond the primary TIS outcome.¹

The most common adverse reactions with brepocitinib were upper respiratory tract infection, headache, fatigue, urinary tract infection, and nausea.¹ Discontinuation due to adverse reactions occurred in 6% of participants treated with brepocitinib 30 mg compared with 11% of those receiving placebo.¹ Lisraya carries a boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events, and thrombosis.¹

These safety findings sit alongside the efficacy data supporting the FDA's Priority Review and Orphan Drug designations for Lisraya.¹ The boxed warning applies across the approved labeling and is consistent with warnings seen with other JAK inhibitors.¹

Brepocitinib's approval gives clinicians a dedicated on-label agent for a population previously managed largely with regimens developed for other diseases.¹ Priovant Therapeutics Inc holds the approval for Lisraya in this indication.¹ The label covers adults with dermatomyositis, reflecting the population enrolled in the pivotal phase 3 trial.¹

References

1. FDA. FDA Approves First Oral Drug Indicated to Treat Dermatomyositis in Adults. August 27, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-drug-indicated-treat-dermatomyositis-adults.

2. ClinicalTrials.gov. A study to evaluate brepocitinib in adult patients with dermatomyositis. NCT05437263. Accessed August 27, 2026. https://clinicaltrials.gov/study/NCT05437263.