FDA Approves First Targeted Therapy for Dermatomyositis
Approval of LISRAYA™ (brepocitinib) marks a significant scientific and clinical milestone for people living with this
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New York, Aug. 27, 2026 (GLOBE NEWSWIRE) — The Muscular Dystrophy Association (MDA) welcomes the U.S. Food and Drug Administration’s approval of LISRAYA™ (brepocitinib), developed and marketed by Priovant Therapeutics, as the first targeted therapy specifically approved to treat adults living with dermatomyositis. The approval represents an important advance for a disease that has historically relied on broad immunosuppressive therapies rather than treatments developed to address its underlying disease biology. Read the company press release here.
“This approval represents meaningful progress for the dermatomyositis community and reflects years of scientific innovation, clinical research, and the commitment of people living with the disease who participated in clinical trials,” said Angela Lek, PhD, Chief Research Officer, Muscular Dystrophy Association. “For people and families living with dermatomyositis, having the first FDA-approved targeted therapy designed specifically for this disease demonstrates the importance of continued investment in neuromuscular research. This milestone reflects the growing momentum toward precision therapies that have the potential to improve outcomes and quality of life for people living with rare neuromuscular diseases.”
Dermatomyositis is a rare autoimmune inflammatory disease characterized by progressive muscle weakness and distinctive skin manifestations. The disease can significantly affect mobility, swallowing, breathing, and quality of life, and may also involve the lungs, heart, and gastrointestinal system. Until now, treatment has largely relied on corticosteroids and other broad immunosuppressive therapies, underscoring the need for treatments that more precisely target the underlying disease process.
“This approval is about so much more than one new treatment—it’s proof that investing in rare disease research changes lives,” said Sierra Allison, MDA community member living with dermatomyositis. “For people living with dermatomyositis, having the first targeted therapy approved by the FDA is a milestone that brings new possibilities and renewed momentum. I’m grateful to everyone who participated in research and clinical trials to help make this day possible, and I hope we continue building on this progress until everyone living with dermatomyositis has access to the treatments they need.”
“The approval of the first targeted therapy for dermatomyositis marks a significant scientific and clinical milestone for a disease that has long had limited treatment options,” said Tahseen Mozaffar, MD, Director of the UCI Health ALS & Neuromuscular Center, an MDA Care Center. “This approval reflects important advances in our understanding of the immune pathways that drive dermatomyositis and demonstrates how those discoveries can be translated into more precise therapies. It also underscores the critical role of rigorous clinical research and the participation of patients in clinical trials in bringing innovative treatment options to people living with rare neuromuscular diseases.”
The approval was supported by findings from the global Phase 3 VALOR clinical trial, the first positive 52-week placebo-controlled study in dermatomyositis, demonstrating clinically meaningful improvements in disease activity and reinforcing the potential of targeted therapies for this rare autoimmune disease.
MDA advances progress for people living with dermatomyositis through research investments, clinical trial infrastructure, education, and partnerships that help accelerate therapeutic development. Through its nationwide network of more than 150 multidisciplinary MDA Care Centers, the organization provides expert, comprehensive care for people living with dermatomyositis and related neuromuscular diseases. People living with dermatomyositis are encouraged to speak with their healthcare providers about whether this newly approved treatment may be appropriate for their care.
About Dermatomyositis
Dermatomyositis is a rare autoimmune inflammatory disease characterized by chronic muscle inflammation, progressive muscle weakness, and distinctive skin rashes. It affects both adults and children and may involve the lungs, heart, gastrointestinal system, and other organ systems. Early diagnosis and multidisciplinary care are essential to improving long-term outcomes and quality of life.
Support and Guidance for Families
For more information on dermatomyositis, including guidance and ongoing support for families and medical professionals, contact the MDA Resource Center by phone 1-833-ASK-MDA1 (1-833-275-6321) or email ResourceCenter@mdausa.org.
Media inquiries contact press@mdausa.org.
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) has been at the center of progress for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions for over 75 years. We unite researchers, clinicians, advocates, and families to speed the pace of discovery, improve access to expert care, and ensure inclusion in every aspect of life. Our mission is simple: give the people we serve the tools and opportunities to live longer, more independent lives. To learn more visit mda.org. Follow MDA on social media on Instagram, Facebook, X, TikTok, LinkedIn, and YouTube.
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Mary Fiance, National Vice President, Strategic Communications Muscular Dystrophy Association press@mdausa.org
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