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Jump to Key Points The FDA has approved brepocitinib (Lisraya), an oral, selective tyrosine kinase 2-JAK1 inhibitor, for the treatment of dermatomyositis, a rare autoimmune disease, in adults.
“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 in the FDA’s Center for Drug Evaluation and Research, said in a statement on August 27. “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.”
Dermatomyositis causes muscle weakness and skin rashes. Brepocitinib is a once-daily tablet that, in blocking JAK pathways, helps reduce harmful inflammation associated with the disease. Current therapies for dermatomyositis such as disease-modifying antirheumatic drugs and intravenous immune globulin are difficult to administer, provide limited efficacy, and are often toxic in their own right.
Brepocitinib was evaluated in a phase 3, double-blind, randomized, placebo-controlled trial, published in The New England Journal of Medicine in March. The study found that the first-in-class drug resulted in significant benefits for adults with dermatomyositis who had not responded to conventional therapies.
- FDA approves first oral dermatomyositis therapy: brepocitinib, adults only.
- Once-daily selective TYK2-JAK1 inhibitor; targets inflammation driving muscle weakness + rash.
- Phase 3 VALOR: 30 mg/day improved Total Improvement Score vs 15 mg + placebo at week 52.
- 30 mg met all 9 key secondary endpoints; adverse event rates similar across groups.
- Boxed warning: serious infection, malignancy, MACE, thrombosis; common AEs included URI, headache, fatigue.
At week 52 of the trial, 81 patients aged 18-75 who were treated with brepocitinib 30 mg daily reached a mean Total Improvement Score of 46.5, compared with a mean of 37.5 in 81 patients taking a 15-mg daily dose and 31.2 in 79 patients in the placebo group. The 30-mg dose of brepocitinib also outperformed placebo in all nine of the study’s key secondary endpoints. Adverse event rates were similar across the three groups (90% for brepocitinib 30 mg, 86% for brepocitinib 15 mg, and 91% for placebo).
Brepocitinib manufacturer Priovant Therapeutics said that in the VALOR trial, the most common adverse reactions for patients taking the drug were upper respiratory tract infection, headache, fatigue, urinary tract infection, nausea, bronchitis, arthralgia, diarrhea, back pain, falls, influenza, and acne.
Discontinuation due to adverse reactions occurred in 6% of patients treated with brepocitinib 30 mg, compared with 11% given a placebo. Brepocitinib carries a boxed warning for serious infections, increased all-cause mortality, cancer, major adverse cardiovascular events, and thrombosis.
The trial was a “landmark study in dermatomyositis,” Ruth Ann Vleugels, MD, MPH, first author of the study report and chair of dermatology at Brigham and Women’s Hospital and professor of dermatology, Harvard Medical School, Boston, said in March at the 2026 annual meeting of the American Academy of Dermatology, as reported by Medscape Medical News. Patients may soon “receive a targeted, effective therapy early in their disease course and avoid systemic corticosteroids and side effects from other traditional DMARDs as well,” she added.
Priovant said that the drug is available immediately in the United States. Full prescribing information can be found here.
Vleugels disclosed relationships with AbbVie, Apogee, AstraZeneca, Boehringer Ingelheim, Bristol Myers Squibb, Eli Lilly, Johnson & Johnson, Pfizer, and Priovant.
Scott Harris has been covering dermatology for more than a decade. He lives near Washington, DC.
