Dermatomyositis Gains First Oral Treatment With Brepocitinib Approval

Victoria Werth, MD, professor of dermatology and rheumatology at the University of Pennsylvania and the Philadelphia VA Medical Center, recently discussed the FDA approval of brepocitinib (Lisraya; Priovant Therapeutics) for dermatomyositis. The approval marks the first on-label oral treatment for the condition, following years reliant on immunosuppressives, prednisone, and infusions. Werth described the approval as an important step for patients previously managed with off-label therapies.¹

The approval was based on the phase 3 VALOR trial (NCT05437263) of 241 adults with dermatomyositis, comparing brepocitinib 30 mg and 15 mg once daily against placebo over 52 weeks.

Oral Option Marks Shift From Off-Label Management

Before this approval, dermatomyositis carried no FDA-approved on-label treatment, Werth said. Patients often relied on immunosuppressives or prednisone for extended periods, along with infusions, to manage disease activity.

"This is revolutionary because up until now, we really didn't have anything on-label for patients, and very often they would have to be on immunosuppressives for very long periods of time, or get prednisone for long periods of time, infusions," Werth said. She added a safe, effective oral option would be especially helpful for patients.

Early Improvement Seen in Refractory Disease

Patients in the trial had not responded well despite background therapy including steroids, Werth said. Steroids can improve symptoms relatively quickly but carry substantial toxicity, limiting their long-term use.

"The fact these patients could start to show improvement as early as four weeks is dramatic," Werth said. In refractory cases, achieving disease control often takes much longer and becomes more difficult, she added.

Objective and Subjective Measures Align

"It's really wonderful when outcome measures incorporate not just the objective disease activity, but also what's subjective for the patient, because the patient's experience is really the most important part," Werth said. Measures including Cutaneous Dermatomyositis Disease Area and Severity Index Activity scores, itch scales, and quality-of-life assessments all improved in the same direction in the trial, she said, which helps establish whether a drug is effective.

Insurance Barriers Complicated Off-Label Access

"It's been really difficult to get off-label drugs for dermatomyositis. It's been considered a bit of an orphan disease," Werth said. Navigating insurance approval for off-label therapies often delayed treatment or blocked it completely, she said.

With an approved option now available, Werth said treatment access should become simpler for patients and clinicians. She said the goal going forward is for patients to start treatment early and reach disease control as quickly as possible.

Reference

1. FDA approves first oral drug indicated to treat dermatomyositis in adults. News release. US Food and Drug Administration. August 27, 2026. Accessed September 8, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-drug-indicated-treat-dermatomyositis-adults