Boston — EMD Serono said the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to enpatoran for the treatment of lupus with active cutaneous manifestations.
EMD Serono is the healthcare business of Merck KGaA, Darmstadt, Germany, in the United States and Canada.
Enpatoran is an oral selective toll-like receptor 7/8 inhibitor designed to modulate pathways central to lupus-related inflammation. The company said the designation could help accelerate development and review of the drug, which is being studied as a potential treatment for skin manifestations of lupus.
“For the 85% of lupus patients whose disease includes skin manifestations, often associated with substantial physical and psychosocial burden, the lack of targeted treatment makes the disease hard to control,” said David Weinreich, MD, MBA, global head of R&D and chief medical officer for the healthcare business of Merck KGaA, Darmstadt, Germany. “This Breakthrough Therapy designation demonstrates that enpatoran has the potential to redefine how we approach lupus, by addressing the visible burden of rash, while potentially driving benefit beyond the skin. We look forward to working with the FDA to potentially bring this much-needed option to patients as quickly as possible.”
The FDA decision was supported by results from the Phase 2 WILLOW study, a multicenter, randomized, double-blind, placebo-controlled, dose-finding trial evaluating the safety and efficacy of orally administered enpatoran.
The trial used a basket and adaptive study design that included patients with cutaneous lupus erythematosus and systemic lupus erythematosus. EMD Serono said the results showed clinically meaningful improvements in lupus symptoms, particularly among patients with active cutaneous manifestations.
Merck KGaA, Darmstadt, Germany, recently began the ELOWEN 1 and ELOWEN 2 studies, a global Phase 3 program evaluating enpatoran’s impact on cutaneous and systemic symptoms in patients with lupus.
Lupus is a chronic autoimmune disease that can affect several organ systems, including the skin, joints, kidneys and central nervous system. Skin manifestations can appear as inflamed, photosensitive lesions on the face, scalp and other areas and may be life-altering.
The company said skin manifestations are the first sign of disease in nearly one-third of lupus cases, but many patients still do not achieve adequate disease control.
