FDA approves brepocitinib for rare skin disease

The FDA has approved Lisraya (brepocitinib) to treat adults with dermatomyositis, rare autoimmune disease. Dermatomyositis affects the skin and muscles and sometimes the lungs, and it can cause chronic inflammation, progressive muscle weakness, and distinctive skin rashes.

“For many decades, the treatment of dermatomyositis has relied on chronic steroids, non-specific immunomodulators, and intravenous immunoglobulin, therapies not targeted to the underlying disease pathobiology,” Ruth Ann Vleugels, M.D., MPH, MBA, Heidi and Scott C. Schuster Distinguished Chair in Dermatology, founding director of the Autoimmune Skin Disease Center and Connective Tissue Disease Clinics at Mass General Brigham, and professor of Dermatology at Harvard Medical School, said in a news release.

Developed by Priovant Therapeutics, Lisraya is a once-daily 30 mg tablet. It is an inhibitor of both TYK2 and JAK1 and is thought to suppress key cytokines linked to autoimmunity. The company did not release information about the wholesale acquisition price but said that patient assistance is available through its program My Compass Support. Patients with commercial insurance may be eligible for a $0 copay.

The approval was based on data from a phase 3 VALOR study that enrolled 241 adults with dermatomyositis. Patients were randomized to receive Lisraya 30 mg once daily, Lisraya 15 mg once daily or placebo for 52 weeks. The study measured Total Improvement Score (TIS) at week 52, a standardized scoring tool that tracks changes across six areas: muscle strength, physical function, skin and other disease activity, muscle enzymes, and both physician and patient assessments of overall health.

Most patients treated with Lisraya were able to achieve both moderate or better improvement on the Total Improvement Score and minimal or no steroid use by the end of the study (55%, compared with 30% on placebo).

Lisraya also demonstrated benefit on independent measures of skin disease and muscle strength based on patients’ own experiences living with dermatomyositis. When asked to rate the overall activity of their disease, patients receiving Lisraya reported more than four times as much improvement as patients on placebo.

On a measure of everyday function, including pain and core activities of daily living such as getting dressed, climbing stairs, and running errands, patients treated with Lisraya achieved clinically meaningful improvement, while those receiving the placebo worsened.

The most common adverse reactions to Lisraya were upper respiratory tract infection, headache, fatigue, urinary tract infection, and nausea. Discontinuation due to adverse reactions occurred in 6% of participants treated with Lisraya 30 mg, compared with 11% of those given a placebo.

Lisraya carries a boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events, and thrombosis.

Data from the VALOR study were presented in March 2026 at the 2026 American Academy of Dermatology Annual Meeting in Denver. The results were published simultaneously in the New England Journal of Medicine (NEJM).

Additional skin-specific secondary endpoints published in JAMA Dermatology in August 2026.