The U.S. Food and Drug Administration has approved Lisraya (brepocitinib) tablets to treat dermatomyositis in adults, making it the first FDA-approved oral treatment for the rare autoimmune disease.

The approval provides a new treatment option for patients who have had limited ways to manage the disease for decades, a news release from the agencysaid. Dermatomyositis causes chronic inflammation, progressive muscle weakness and distinctive skin rashes as the immune system attacks the muscles and skin.

“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, M.D., director of the Office of Immunology and Inflammation in the FDA’s Center for Drug Evaluation and Research, said in the release. “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.”

Lisraya is a once-daily oral tablet that works as a Janus kinase (JAK/TYK2) inhibitor. By blocking JAK pathways, which play a key role in the body’s immune and inflammatory responses, the drug helps reduce harmful inflammation that damages muscles and skin.

The efficacy and safety of Lisraya were evaluated in a phase 3 randomized, double-blind, multicenter, placebo-controlled study, NCT05437263, involving 241 adults with dermatomyositis. Participants were randomly assigned to receive Lisraya, also known as brepocitinib, at 30 mg once daily, brepocitinib at 15 mg once daily or a placebo for 52 weeks.

According to the release, researchers assessed participants using the Total Improvement Score at week 52. The standardized measure tracks changes across six areas: muscle strength, physical function, skin and other disease activity, muscle enzymes and physician and patient assessments of overall health. The score is used to assess the extent to which a patient’s dermatomyositis has improved.

Patients who received Lisraya 30 mg had a higher average Total Improvement Score at week 52 than those who received a placebo, indicating a greater clinical response and improved disease control. The Lisraya group also showed improvements in physical function and skin disease activity and was more likely to reduce corticosteroid use by week 48.

The agency said most common adverse reactions associated with Lisraya included upper respiratory tract infection, headache, fatigue, urinary tract infection and nausea. Adverse reactions led to treatment discontinuation in 6% of participants who received Lisraya 30 mg compared with 11% of those who received a placebo.

Lisraya carries a boxed warning for serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events and thrombosis. The warnings address the risks of serious infections, a higher risk of death from any cause, cancers, serious heart and blood vessel problems and blood clots.

The FDA granted Lisraya Orphan Drug and Priority Review designations. Priovant Therapeutics Inc. received the approval for Lisraya as a treatment for dermatomyositis.