Merck announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to enpatoran for the treatment of lupus with active cutaneous manifestations. Enpatoran is an oral selective toll-like receptor (TLR) 7/8 inhibitor, designed to modulate pathways central to lupus-related inflammation.

David Weinreich, MD, MBA, Global Head—R&D and Chief Medical Officer (CMO)—Healthcare Business, Merck. For the 85 percent 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. 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 Breakthrough Therapy Designation is supported by results from the phase 2 WILLOW study, a multicenter, randomised, double-blind, placebo-controlled, dose-finding study to evaluate the safety and efficacy of orally administered enpatoran. The trial used an innovative basket and adaptive study design including patients with both Cutaneous Lupus Erythematosus (CLE) and Systemic Lupus Erythematosus (SLE). The results demonstrated that enpatoran delivered clinically meaningful improvements of lupus symptoms, particularly in patients with active cutaneous manifestations.

Additionally, Merck recently initiated the ELOWEN 1 and ELOWEN 2 studies, a global phase 3 program evaluating the therapy’s impact on both cutaneous and systemic symptoms in patients with lupus and potential links between skin and systemic disease activity.

Lupus is a chronic autoimmune disease that can affect multiple organ systems, including the skin, joints, kidneys, and Central Nervous System (CNS). Skin manifestations, which can present as inflamed, photosensitive lesions on the face, scalp and other areas, may be life-altering and are sometimes irreversible on their own. But, despite skin manifestations being the first sign of disease in nearly one-third (29 percent) of lupus cases, many patients still do not achieve adequate disease control.

FDA Breakthrough Therapy Designation is a process aimed at expediting the development and review of drugs for serious conditions, where the preliminary clinical evidence indicates that the drug may offer a substantial improvement over available therapy.