SILVER SPRING, MARYLAND, Aug 27, 2026
Priovant Therapeutics Inc. has received U.S. Food and Drug Administration (FDA) approval for Lisraya (brepocitinib) tablets for the treatment of dermatomyositis in adults, providing the first FDA-approved oral treatment option specifically indicated for the rare autoimmune disease. The approval addresses a longstanding unmet medical need for patients who have historically relied on therapies developed for other inflammatory and autoimmune conditions. Lisraya is administered as a once-daily oral tablet and is designed to help control the inflammation responsible for muscle weakness and characteristic skin manifestations associated with dermatomyositis.
Lisraya Targets JAK/TYK2 Inflammatory Pathways
Dermatomyositis is a rare autoimmune disease in which the immune system attacks the muscles and skin, resulting in chronic inflammation, progressive muscle weakness and distinctive skin rashes. The disease can significantly affect physical function and quality of life, while treatment options have historically been limited. Lisraya (brepocitinib) is a Janus kinase (JAK)/TYK2 inhibitor designed to interfere with signaling pathways involved in immune and inflammatory responses. By blocking these pathways, the therapy is intended to reduce the inflammatory activity contributing to muscle and skin damage. The FDA approval therefore introduces an oral, targeted treatment approach for adults with dermatomyositis and expands the therapeutic landscape for a disease with limited approved treatment options.
Phase 3 Study Demonstrates Clinical Benefit
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 randomized to receive brepocitinib 30 mg once daily, brepocitinib 15 mg once daily, or placebo over a 52-week treatment period. The study assessed Total Improvement Score (TIS) at Week 52, a standardized measure incorporating multiple aspects of dermatomyositis, including muscle strength, physical function, skin and other disease activity, muscle enzymes, and physician and patient assessments of overall health. Patients treated with Lisraya 30 mg demonstrated a higher average TIS score at Week 52 than those receiving placebo, indicating greater clinical response and disease control. The treatment group also demonstrated improvements in physical function and skin disease activity, while patients receiving the 30 mg dose were more likely to reduce corticosteroid use by Week 48. These findings supported the FDA’s decision to authorize Lisraya for adult dermatomyositis.
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Safety Profile and FDA Designations
The most common adverse reactions reported with Lisraya included upper respiratory tract infection, headache, fatigue, urinary tract infection and nausea. Discontinuation because of adverse reactions occurred in 6% of participants receiving Lisraya 30 mg, compared with 11% of participants receiving placebo. Because JAK pathway inhibition can be associated with serious risks, Lisraya carries a boxed warning covering serious infections, increased all-cause mortality, malignancies, major adverse cardiovascular events and thrombosis. The safety profile will therefore remain an important consideration in treatment decisions and patient monitoring. The FDA previously granted Lisraya Orphan Drug and Priority Review designations for the dermatomyositis indication, reflecting the rarity of the disease and the need for new therapeutic options. With the August 27 approval, Priovant Therapeutics gains authorization for a once-daily oral therapy specifically indicated for adults with dermatomyositis, representing a notable milestone in the development of treatments for rare autoimmune diseases. The approval also highlights the continued clinical development of targeted immunomodulatory therapies aimed at controlling disease activity while potentially reducing reliance on corticosteroids.
Source: Priovant Therapeutics press release
