The U.S. Food and Drug Administration approved Lisraya, or brepocitinib, on August 27 as the first oral treatment specifically indicated for adults with dermatomyositis. The once-daily 30-milligram tablet gives patients with the rare autoimmune disease a targeted option after decades in which care often depended on corticosteroids, broad immune-suppressing medicines and intravenous immunoglobulin.
Dermatomyositis occurs when the immune system attacks muscle and skin, producing chronic inflammation, progressive weakness and distinctive rashes that can be painful or itchy. The systemic illness can restrict basic activities such as dressing, climbing stairs and running errands. The FDA said brepocitinib inhibits the JAK and TYK2 pathways involved in immune and inflammatory signaling, thereby reducing inflammation that damages muscle and skin.
The decision rested on VALOR, a randomized, double-blind, multicenter phase 3 trial involving 241 adults whose disease had resisted previous therapy. Participants received 30 milligrams of brepocitinib, 15 milligrams or placebo once daily for 52 weeks while permitted background treatments continued and glucocorticoids tapered. Investigators assessed improvement with a composite score covering muscle strength, physical function, skin and other disease activity, muscle enzymes, and patient and physician evaluations.
At week 52, the average Total Improvement Score reached 46.5 in the approved 30-milligram group, compared with 31.2 for placebo, according to the published trial results. The 30-milligram dose also performed better across nine key secondary measures, and benefits appeared as early as week four. Roivant reported that 55 percent of treated patients achieved at least moderate improvement with minimal or no steroid use, compared with 30 percent receiving placebo.
The medicine carries important risks. Common adverse reactions included upper respiratory infections, headache, fatigue, urinary tract infections and nausea. Serious infections occurred in 10 percent of patients receiving 30 milligrams and 1 percent of those on placebo during the trial. Its boxed warning also covers serious infections, malignancies, major cardiovascular events, thrombosis and increased mortality, and it is not recommended with other JAK or TYK2 inhibitors or biologic disease-modifying drugs.
The FDA granted the medicine Priority Review and Orphan Drug designations before approval, reflecting both the seriousness and rarity of the condition. Roivant said prescriptions are immediately available in the United States through specialty pharmacies. The approval expands choice but does not remove the need for clinicians to weigh infection, cardiovascular, cancer and clotting risks, monitor patients carefully and decide whether the drug should replace or supplement existing treatment.
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