By Sangmee Bae, MD as told to Kendall Morgan
Sangmee Bae, MD, is a rheumatologist at UCLA Health in Los Angeles. She treats patients with dermatomyositis and studies various forms of myositis and related health conditions, including interstitial lung disease. The information here reflects her clinical expertise and experience. Bae was not involved in the development of brepocitinib (Lisraya), which became the first oral medicine for adults with dermatomyositis following positive phase 3 clinical trial results and the medicine's approval by the FDA in August 2026.
Dermatomyositis is a subgroup of many different inflammatory myopathies, which are diseases affecting skeletal muscles. It causes muscle weakness and skin rashes, which can be debilitating. In the past, this condition has been underrecognized compared to other autoimmune conditions, like lupus or rheumatoid arthritis. That's because myositis is a group of rare, or "orphan," diseases, says Bae.
As a result, people with this condition are frequently misdiagnosed. There has been an unmet need for approved treatments that specifically target the inflammation that causes this condition. We spoke with Bae to learn more about how this medication works and who it will benefit most.
How will brepocitinib (Lisraya) change dermatomyositis treatment?
Brepocitinib (Lisraya) is approved for adults with dermatomyositis. Historically, people with dermatomyositis would often get treated with steroids. In general, it is a very steroid-responsive disease. The problem with that is patients often do OK initially with high doses of steroids. But, as time goes on, and we try to bring the steroids down, the disease would come right back. Prolonged use of glucocorticoids also comes with safety concerns. Conventional treatments really didn't address the underlying pathology of the muscle disease, and the glucocorticoids alone wouldn't put patients into remission.
Later, doctors started implementing disease-modifying drugs used in other rheumatic diseases to treat dermatomyositis. Intravenous immunoglobulin (IVIG) is an infusion medicine that's approved for dermatomyositis, but it's an expensive drug and requires an infusion. The approval of IVIG in 2021 was a game changer for treating dermatomyositis. However, we started finding patients with disease that was tough to treat. They didn't respond, and we could never get them into remission. Even with IVIG, they had poor quality of life. We started resorting to more aggressive drugs with more potential side effects. While this new medicine can be taken orally and may benefit all patients with this disease, people whose disease was recalcitrant, meaning it didn't respond to other treatments, stand to benefit the most from this new treatment option.
What research led to its discovery?
Advances in treating dermatomyositis came out of an effort by researchers to classify this disease as a separate entity. It's not a sister disease of lupus anymore, but it really is its own disease entity. They had to define its characteristics and the best ways to measure it so that researchers could actually conduct clinical trials. It involved an initiative to develop myositis outcome measures and new classification criteria. That was the first step.
The research also started going into more detailed studies to find what really drives the disease and identify promising drug targets. One of the targets that was identified in dermatomyositis is the interferon signature. Overactivation of this immune pathway is a defining feature of the disease underlying the inflammation and other symptoms in the skin disease, and also in the muscle. As a result, it made sense to try a JAK inhibitor.
What sets brepocitinib apart from other JAK inhibitors, and what does its approval mean for people with this condition?
JAK inhibitors are a class of medicines that block signals driving inflammation to calm the immune system. Small studies first started looking at JAK inhibitors approved for other conditions in dermatomyositis. These medicines were being used off-label to treat recalcitrant disease. But there was no JAK inhibitor approved for dermatomyositis until now. Brepocitinib is a once-daily pill that specifically inhibits both JAK1 and TYK2 to reduce signals that drive inflammation in dermatomyositis. So the specific target is different from other JAK inhibitors.
Even though dermatomyositis is a skin disease, some people with this condition can't sleep. They may itch their scalp until they bleed. These are terrible skin diseases that really impact quality of life. That was the reason to study JAK inhibitors and, as a physician that sees dermatomyositis patients, it's wonderful to see an FDA indication for any drug, but then also for a drug that is proven to be helpful for the skin and muscle disease. We were all excited when the [trial] data came out.
How fast does brepocitinib work?
One of the big findings in the trial was not just the primary efficacy, but how fast the patients [in the trial] responded to the drug. I think what sets brepocitinib apart from other existing options, including IVIG and all the conventional drugs, is the hope that it will work faster.
I usually have told my patients that they might see a little bit of difference in the first month or two, maybe three months. Usually, I assess efficacy after at least three months of therapy. But the phase 3 clinical trial results for brepocitinib start to show efficacy even in month one, which is much faster than other medications. The trial didn't assess the primary endpoint until the end of the study, but the decline in disease activity is quite rapid, which is very encouraging.
How does taking brepocitinib compare to IVIG?
Brepocitinib is an oral drug. IVIG is intravenous. It's an infusion that requires you to sit in an infusion center or at home for three to six hours each time, and it's multiple infusions a month. It can sometimes be difficult to get it paid for. Many people with dermatomyositis are young and working. You have to find time off of work to do the infusions. After the infusion, a substantial number of people have headaches, especially if they try to rush the infusion because they don't have time. The side effects of IVIG can be a major limitation. With an oral medication, you don't have those logistical constraints.
Are there side effects?
The trial reported some serious adverse events in those taking brepocitinib. The main one was an increased risk of serious infections [10% in the brepocitinib group versus 1% in the placebo group].
Are there people who shouldn't take this treatment?
JAK inhibitors as a class have safety concerns, including blood clots and increased risk for cardiovascular disease, particularly in those with existing cardiovascular risk factors. If you've had a prior heart attack or stroke or you have uncontrolled diabetes, high blood pressure, or a strong family history of cardiovascular disease at young ages, I would assume the same warnings that exist for other JAK inhibitors would apply. However, in the trial, they didn't see any more cardiovascular events in those receiving brepocitinib after 52 weeks.
If I had a patient that really struggled with recurrent infections in the past, I would think twice about putting them on brepocitinib. If they just had a stroke or blood clot, and there was uncertainty about their future risk of recurrent clots, I would hold off until we have a little bit more data. As with any new drug, more long-term study is needed. That said, I have already had a couple of people who haven't achieved remission with other medications or who were having trouble with side effects ask me if they could start taking this new medication. Ever since we learned that the clinical trial results were positive, I have been telling my patients that this was coming.
Will insurance cover it?
It's always a good idea to check with your health insurance to confirm coverage. It may require prior authorization. This is a brand-new FDA approval, but I'm hoping that it should sail through.
What's the best way for people with dermatomyositis to find out more?
They should go talk to their own doctor or provider. Especially if you've tried conventional treatment and have recalcitrant disease or can't get IVIG, then I would certainly suggest reaching out to your care team. People can try it and see how they do. In general, JAK inhibitors are pretty well tolerated.
What are you especially excited to learn as people get access to this new medicine?
I'm excited to know how well the trial results will translate in the real world and especially whether the disease will really respond that quickly. I'm also curious about how it will work for other aspects of this disease. The emphasis in the trial was really on the improvement of skin and muscle disease. The effect on other myositis symptoms has not been tested. For example, lung disease is a very substantial part of dermatomyositis as well. Twenty percent of patients have some lung disease, but none of the outcome measures focused on lung improvement. Assessing those other aspects will require future studies and real-world experience.
