Kiniksa Pharmaceuticals Receives U.S. Orphan Drug Designation For KPL-387 To Treat Pericarditis

Kiniksa Pharmaceuticals International, plc, a biopharmaceutical company focused on developing and commercializing novel therapies for diseases with unmet medical needs, particularly in cardiovascular indications, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to KPL-387 for the treatment of pericarditis, including recurrent pericarditis. KPL-387 is an independently developed monoclonal antibody that targets the human interleukin-1 receptor 1 (IL-1R1), blocking the signaling activity of the cytokines interleukin-1α (IL-1α) and interleukin-1β (IL-1β).

John F. Paolini, M.D., Ph.D., FACC, Chief Medical Officer of Kiniksa, stated that the FDA’s designation underscores the company’s commitment to addressing the needs of patients with this rare and debilitating condition. He highlighted that KPL-387 has the potential to offer an additional treatment option through a convenient monthly subcutaneous self-injection in a liquid formulation. Data from the Phase 2 dose-focusing portion of the KPL-387 Phase 2/3 trial for recurrent pericarditis are expected in the second half of 2026.