WASHINGTON, D.C. — The Food and Drug Administration approved Priovant Therapeutics Inc.’s Lisraya for adults with dermatomyositis, providing an oral treatment for the rare autoimmune disease after a Phase 3 trial showed improved disease control and reduced corticosteroid use.
Lisraya, or brepocitinib, is a once-daily tablet that inhibits Janus kinase pathways involved in immune and inflammatory responses. Dermatomyositis causes the immune system to attack muscles and skin, resulting in chronic inflammation, progressive muscle weakness and characteristic rashes.
The approval gives patients a treatment specifically evaluated for dermatomyositis after years in which therapeutic options have been limited and patients have often relied on drugs used for other diseases.
“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, director of the FDA’s Office of Immunology and Inflammation, stated.
The FDA based its decision on a randomized, double-blind Phase 3 study involving 241 adults with dermatomyositis. Participants received either 30 milligrams of brepocitinib once daily, 15 milligrams once daily or a placebo over 52 weeks.
Patients receiving the approved 30-milligram dose recorded a higher average Total Improvement Score at 52 weeks than those receiving placebo, indicating a greater clinical response, according to the FDA.
The scoring system measures changes in six areas, including muscle strength, physical function, skin and other disease activity, muscle enzymes and physician and patient assessments of overall health.
Patients taking the 30-milligram dose also showed improvements in physical function and skin disease activity and were more likely to reduce their use of corticosteroids by week 48.
The drug carries significant safety warnings. Its boxed warning covers serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events and thrombosis.
Among the most commonly reported adverse reactions were upper respiratory tract infections, headaches, fatigue, urinary tract infections and nausea. Six percent of patients receiving the 30-milligram dose discontinued treatment because of adverse reactions, compared with 11% of placebo recipients.
The FDA granted Lisraya Orphan Drug and Priority Review designations before approving the treatment.
Support the local news that supports Chester County. MyChesCo delivers reliable, fact-based reporting and essential community resources—free for everyone. If you value that, click here to become a patron today.