FDA Approves Priovant’s Lisraya as First Oral Therapy for Rare Autoimmune Muscle Disease

Rare Daily Staff

The U.S. Food and Drug Administration approved Lisraya, a once-daily pill for adults with dermatomyositis, a rare autoimmune disease that can cause debilitating muscle weakness and skin rashes.

The decision gives patients the first FDA-approved oral treatment specifically indicated for the condition.

Dermatomyositis occurs when the immune system mistakenly attacks muscle and skin tissue, causing inflammation, progressive weakness and often a distinctive rash. People with the disease have long had limited treatment options and may rely on medicines developed for other conditions.

Lisraya is designed to reduce that immune-driven inflammation. It blocks signaling pathways known as JAK and TYK2, which help regulate immune and inflammatory responses. The medicine is taken as a -milligram tablet once a day. The FDA previously granted orphan drug and priority review designations.

The FDA based its decision in part on a phase clinical trial involving adults with dermatomyositis. Participants received either milligrams of Lisraya daily, a lower -milligram dose of brepocitinib or a placebo for weeks.

Patients receiving the approved -milligram dose had a higher average Total Improvement Score at one year than those who received placebo. The score incorporates six measures of disease status, including muscle strength, physical function, skin activity, muscle-enzyme levels, and physician and patient assessments of overall health.

The FDA said patients on Lisraya also showed improvements in physical function and skin disease activity. They were more likely than placebo recipients to reduce their use of corticosteroids by week , an important consideration because long-term steroid use can carry substantial side effects.

The approval comes with a boxed warning, the FDA’s most prominent safety warning, for risks that include serious infections, cancer, major cardiovascular events, blood clots and an increased risk of death from any cause.

The most commonly reported side effects were upper respiratory tract infections, headache, fatigue, urinary tract infection and nausea. In the pivotal study, percent of patients taking the -milligram dose stopped treatment because of adverse reactions, compared with percent of patients given placebo.

“For too long, patients with dermatomyositis have faced a significant unmet need for effective treatments,” Nikolay Nikolov, director of the Office of Immunology and Inflammation in the FDA’s drug center, said in a statement. He called the approval “a meaningful step forward” for patients and their health care providers.