Kiniksa Pharmaceuticals Announces U.S. Orphan Drug Designation for KPL-387 for the Treatment of Pericarditis

Core Viewpoint - Kiniksa Pharmaceuticals has received Orphan Drug Designation from the FDA for KPL-387, aimed at treating pericarditis, including recurrent cases, which highlights the company's commitment to addressing unmet medical needs in rare diseases [1][2]. Company Overview - Kiniksa Pharmaceuticals is focused on developing and commercializing novel therapies for diseases with unmet needs, particularly in cardiovascular indications [4]. - The company aims to improve the lives of patients suffering from debilitating diseases through innovative treatments [4]. Product Information - KPL-387 is a fully human IgG2 monoclonal antibody that targets human IL-1R1, inhibiting the activity of cytokines IL-1α and IL-1β [5]. - The drug is expected to offer a convenient treatment option for recurrent pericarditis patients, potentially allowing for a single monthly subcutaneous self-injection [2][5]. - Data from the Phase 2 dose-focusing portion of the KPL-387 Phase 2/3 trial is anticipated in the second half of 2026 [2]. Orphan Drug Designation - The FDA's Orphan Drug Designation is granted to products treating rare diseases, which includes those affecting fewer than 200,000 people in the U.S. [3]. - This designation provides financial incentives such as grant funding for clinical trials, tax advantages, and waivers for user fees [3].