MyoCon! September 24–27, 2026.
If you’ve been hearing cheers from TMA today, there’s a good reason. We are absolutely thrilled to share news that many patients, care partners, clinicians, and researchers have been working toward for years: the US Food and Drug Administration (FDA) has approved Lisraya™ (brepocitinib) for the treatment of adults with dermatomyositis (DM). This approval marks the first oral therapy specifically indicated for dermatomyositis and the first targeted therapy approved for this rare autoimmune disease.
For those of us in the myositis community, this is more than just another regulatory announcement. For many patients, treatment options have typically relied on corticosteroids and medicines originally developed for other diseases. Therapies like intravenous immunoglobulin can help, although many patients continue to struggle with disease activity and treatment-related side effects.
Today’s approval changes that treatment landscape. Lisraya is a once-daily oral medication that works by targeting immune pathways known as JAK and TYK2, helping reduce the inflammation that drives the disease.
The FDA’s decision was based on results from a Phase 3 clinical trial involving 241 adults with dermatomyositis. Participants receiving the approved 30 mg dose experienced greater overall improvement in disease activity compared with those receiving placebo. Researchers also reported improvements in physical function and skin disease activity, and patients were more likely to reduce their use of corticosteroids.
These results are especially encouraging because they address multiple aspects of DM, including both muscle and skin symptoms, while offering the possibility of reducing long-term reliance on steroids.
A Victory Decades in the Making
Anyone who has lived with DM understands that progress in rare disease research doesn’t happen overnight. Every clinical trial participant, every researcher, every physician, every advocate, and every donor who has supported myositis research has helped make this day possible. The approval of Lisraya is a reminder that when patients, investigators, industry partners, and advocacy organizations work together, meaningful advances can happen.
And make no mistake: this is a meaningful advance. For decades, members of our community have hoped for treatments designed specifically for myositis rather than borrowed from other conditions. Today marks an important step toward that goal.
What This Means for Patients
An FDA approval does not mean DM has been cured. DM is a complex disease with varied symptoms and response to treatments. No single treatment works for everyone. Patients should always talk with their healthcare providers about whether any therapy is appropriate for their individual situation. But today’s news means that adults with dermatomyositis now have hope: a new FDA-approved treatment option specifically studied in their disease.
“TMA congratulates Priovant Therapeutics and extends our deepest gratitude to the patients, families, and investigators who made this approval possible by participating in the clinical trial that established Lisraya’s safety and efficacy,” said Paula J. Eichenbrenner, MBA, CAE, TMA executive director. “We are very excited that this new therapy has been approved—and that scientific advances continue to benefit people diagnosed with dermatomyositis and other forms of myositis.”
Today, we are absolutely jumping up and down for joy. And tomorrow, we will continue working toward the next breakthrough. Because one approval is wonderful, but a future with many more is even better.
For more information about Lisraya (brepocitinib), patients should consult their healthcare providers and review the FDA-approved prescribing information. [ebs.publicnow.com]
That’s good news. What about Car T Cell therapy?
Reading everything about that sounds great. How soon will that have FDA approval?
Hello Kathy. CAR T cell therapy is still in clinical trials with several different companies. This is a years long process, and we don’t have a good way of knowing when the trials will be completed, what the results might be, and whether the companies will be able to apply for FDA approval. Have you thought about enrolling in one of the trials?
Cart-T testing was just cancelled by the FDA because of all the side effects. There is another medicine coming out next year. Efgartigimod.
It’s true that last week two companies put their clinical trials on hold for CAR T therapy in autoimmune disease. They have not cancelled them completely, and there are several other companies that are still testing some form of CAR T treatment. So there is still hope for
That’s wonderful news. Thank you for sharing this information and finally giving us with dm some hope for treatment options. A huge thank you to the countless physicians, researchers, scientists and entire healthcare and science community to their relentless research efforts. We appreciate it more than you will ever know ❤️
What does this approval mean for patients in Europe?
This is a great question. If a medication is approved by the U.S. FDA, it does not automatically mean it is approved in Europe. The U.S. and Europe have separate regulatory systems and make independent decisions. The company will need to submit (or possibly already has) a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA). EMA is like the FDA and will review the application and approve or not. We expect this to be easily approved and available soon in Europe too.
Is this treatment an option for patients with JDM specifically?
Right now, the approval is only for adult DM. It was only tested in adults.
Does it also treat ILD?
What we know is that brepocitinib demonstrated broad efficacy in adult dermatomyositis, improving overall disease activity, muscle strength, skin disease, physical function, and steroid reduction in the Phase 3 VALOR trial. ILD was not something they tested for. The FDA approval is for adult dermatomyositis, not for ILD.
when can we expecct this to be available , when can we start asking our dr’s about it ?
Angela, Lisraya is now available for doctors to prescribe. You can definitely speak with your doctor about whether this is an option for you.
I’ve been on imuran for 15 years And developed melanoma. My neurologist/ rheumatologist both said to wean off the Imuran. I also take plaquinol for Lupus and there was suspicion my eyes were being affected so they cut me down on that as well. I have DM symptoms, popping up, severe fatigue, pem, and my muscles in my thighs and arms are definitely being affected already. Already. My Labs don’t show any information yet. I asked what is the plan and they said there’s no medication available for me because of the skin cancer. I specifically asked about Lisraya And was told I’m not a candidate. Could you help me understand why since I can’t get an explanation that’s clear from my doctors.
Janice, I’m so sorry you’re going through this. We have a webinar tomorrow where you can ask this question of the speakers. https://www.myositis.org/event/dm-treatment-webinar-priovant/
What is the difference between this and xeljanz?
(Tofacitinib))
Brepocitinib inhibits TYK2 and JAK1 pathways in the immune system, which more directly target the biology driving dermatomyositis.
Tofacitinib (Xeljanz) primarily inhibits JAK1 and JAK3, with some activity against JAK2, but has minimal TYK2 activity. This gives a more general response against autoimmune diseases.