News Release

PHILADELPHIA – An experimental drug called litifilimab (known as BIIB059) could be a powerful long-term treatment for cutaneous lupus erythematosus (CLE), a form of lupus that occurs in the skin and can severely impact quality of life, according to a study from the Perelman School of Medicine at the University of Pennsylvania and the Corporal Michael J. Creszcenz VAMC. The study drug was able to decrease CLE activity in the skin. The results, published in the New England Journal of Medicine, are promising since current treatments for the disease are not always very effective.

“Some patients in our trial saw huge improvements within a month,” said Victoria Werth, MD, a professor of Dermatology at Penn and the chief of Dermatology at the Corporal Michael J. Creszcenz VAMC. “The study showed the drug decreased CLE activity relative to a placebo.”

CLE can occur both in the presence of or absence of systemic lupus erythematosus (SLE).

“CLE can cause scarring, atrophy in the skin, and skin discoloration, as well as permanent hair loss. These impacts on physical appearance also affect mental and emotional health and well-being.

The study ran for 16 weeks with 132 participants. Improvements began to appear within weeks, with the most impactful improvements seen at the 12 and 16-week mark.

Existing therapies for CLE, including antimalarial medications like hydroxychloroquine, immunosuppressives, and steroids have had limited or inconsistent effectiveness or frequent side effects. Litifilimab, however, works differently. As part of a class of drug therapies known as humanized monoclonal antibodies, litifilimab is an antibody that targets the blood dendritic cell antigen 2 (BDCA2) receptor on plasmacytoid dendritic cells, leading to downregulation of pro-inflammatory proteins that are believed to be key culprits behind the damaging inflammation associated with CLE.

“Not only do the available drugs not always work, but they are also not always well tolerated,” said Werth. “Sometimes, a patient may have to move up to immunosuppressants, and those have a lot of side effects like higher risk of infection. So being able to develop a different approach could be pivotal.”

This multisite trial was the first successful randomized phase 2 trial of any new CLE-specific systemic treatment. No new drugs have been approved specifically for CLE for over 50 years, and the few available are used off label or were grandfathered into use. According to Werth, a key to the trial’s success was the Cutaneous Lupus Erythematosus Disease Area and Severity Index-Activity (CLASI-A), a measurement tool Penn Medicine researchers helped develop. Readings in 13 skin regions generate CLASI-A scores ranging from 0 to 70, with higher scores indicating more widespread or severe disease. The tool empowered investigators to more precisely and accurately characterize CLE disease activity including hair loss.

With these promising results, the company Biogen, which funded the study and developed litifilimab, will likely take this medication to a further trial, according to Werth.


Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, excellence in patient care, and community service. The organization consists of the University of Pennsylvania Health System and Penn’s Raymond and Ruth Perelman School of Medicine, founded in 1765 as the nation’s first medical school.

The Perelman School of Medicine is consistently among the nation's top recipients of funding from the National Institutes of Health, with $550 million awarded in the 2022 fiscal year. Home to a proud history of “firsts” in medicine, Penn Medicine teams have pioneered discoveries and innovations that have shaped modern medicine, including recent breakthroughs such as CAR T cell therapy for cancer and the mRNA technology used in COVID-19 vaccines.

The University of Pennsylvania Health System’s patient care facilities stretch from the Susquehanna River in Pennsylvania to the New Jersey shore. These include the Hospital of the University of Pennsylvania, Penn Presbyterian Medical Center, Chester County Hospital, Lancaster General Health, Penn Medicine Princeton Health, and Pennsylvania Hospital—the nation’s first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is an $11.1 billion enterprise powered by more than 49,000 talented faculty and staff.

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