The Lupus Research Alliance welcomes the decision by the U.S. Food and Drug Administration (FDA) to grant Breakthrough Therapy designation to enpatoran in clinical development by EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, for the treatment of lupus with active cutaneous manifestations. The FDA grants this designation to expedite the review of drugs for serious conditions that may demonstrate substantial improvement over available therapies.
Cutaneous manifestations (skin-related symptoms) in lupus vary, but can include butterfly rashes, ring-shaped sores, or red, scaly patches on the skin. These manifestations affect approximately 70-85% of people with lupus, and may occur as part of systemic lupus erythematosus (SLE) or independently as cutaneous lupus erythematosus (CLE).
“There is major unmet need in treatments for skin manifestations of lupus,” Albert T. Roy, President & CEO of the Lupus Research Alliance, said. “It is encouraging to see progress in the development and recognition by the U.S. FDA to help fill that gap.”
As reported in the July 8 press release from EMD Serono, enpatoran is an investigational, oral, selective inhibitor of toll-like receptors 7 and 8 (TLR7/8). Merck also reported that the FDA’s Breakthrough Therapy designation was supported by results from the Phase 2 WILLOW study, which demonstrated clinically meaningful improvement in lupus symptoms, particularly among patients with active cutaneous manifestations.
The Lupus Research Alliance funded early research on the link between toll-like receptors and inflammation. Lupus Therapeutics, the clinical affiliate of the Lupus Research Alliance, worked with EMD Serono to bring the patient perspective to drug development. Lupus Therapeutics is now supporting the Phase 3 ELOWEN 1 and ELOWEN 2 studies evaluating the impact of enpatoran on both cutaneous and systemic symptoms in patients with lupus and potential links between skin and systemic disease activity.
EMD Serono is a member of the Lupus Accelerating Breakthroughs Consortium (Lupus ABC), convened by the Lupus Research Alliance as a public-private partnership with the FDA that focuses specifically on advancing the development of safer and more effective treatments urgently needed for people with lupus. A dedicated working group of this consortium focuses on the use of the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) as a primary outcome measure for CLE clinical trials.
Additional information about the designation is available via the media release from EMD Serono.
About Lupus
Lupus is a chronic, complex autoimmune disease that affects millions of people worldwide. In lupus, the immune system, meant to defend against infections, produces autoantibodies that mistake the body’s own cells as foreign, causing other immune cells to attack organs such as the kidneys, brain, heart, lungs and skin, as well as blood and joints. Ninety percent of people with lupus are women, most often diagnosed between the ages of 15–45. Black, Latinx, Indigenous, Asian and Pacific Islander people are disproportionately affected by lupus.
About the Lupus Research Alliance
The Lupus Research Alliance is the largest non-governmental, non-profit funder of lupus research worldwide. The organization aims to transform treatment by funding the most innovative lupus research, fostering scientific talent, and driving discovery toward better diagnostics, improved treatments and, ultimately, a cure for lupus. Because the Lupus Research Alliance’s Board of Directors funds all administrative and fundraising costs, 100% of all donations go to support lupus research programs.
For more information or to donate to lupus research, visit the LRA at LupusResearch.org and on social media at: X, Facebook, LinkedIn, and Instagram.
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Media Contact:
Margy Meislin
mmeislin@lupusresearch.org
Tags: U.S. Food and Drug Administration, CLE, enpatoran, Merck KGaA, cutaneous, skin lupus, treatment, clinical trials, EMD Serono, FDA