Handok's Empaveli (pegcetacoplan) has been granted health insurance coverage in Korea, marking a significant advancement in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Starting Friday, the reimbursement will enable broader access to this innovative therapy for adult PNH patients. Empaveli, developed by Swedish Orphan Biovitrum (Sobi) and Apellis Pharmaceuticals, is the first C3-targeted treatment designed to address both intravascular and extravascular hemolysis in PNH. This approval addresses a critical unmet need, as existing treatments primarily target intravascular hemolysis.
Clinical Efficacy and Trial Data
Empaveli's approval is supported by robust clinical trial data. The phase 3 PEGASUS trial demonstrated Empaveli's superiority over eculizumab in improving hemoglobin concentration over 16 weeks. An extension study showed that patients treated with Empaveli maintained lactate dehydrogenase (LDH) levels, a key indicator of intravascular hemolysis, at less than 1.5 times the upper normal limit for 48 weeks. Notably, 85% of patients in the pegcetacoplan group required no transfusions over 16 weeks, compared to only 15% in the eculizumab group. Furthermore, quality of life assessments, measured by FACIT-fatigue scores, revealed a 9.2-point improvement in the pegcetacoplan group from baseline, while the eculizumab group experienced a 2.7-point decrease, resulting in an 11.9-point difference.
The phase 3 PRINCE trial, which observed PNH patients with no prior complement inhibitor treatment over 26 weeks, further validated pegcetacoplan's efficacy. In this study, 85.7% of patients treated with pegcetacoplan achieved stable hemoglobin levels, and their LDH levels remained below the upper normal limit.
Addressing Unmet Needs in PNH Treatment
PNH is a rare, chronic, and life-threatening blood disorder characterized by the destruction of red blood cells due to an acquired mutation that activates the complement system. Until now, the only PNH treatment covered by health insurance in Korea was a C5 inhibitor, which targets only intravascular hemolysis. Studies in the U.S. and Japan have indicated that a significant proportion of patients treated with C5 inhibitors (eculizumab, ravulizumab) continue to experience persistent fatigue and symptoms like shortness of breath, highlighting the need for therapies that address extravascular hemolysis.
Reimbursement Criteria and Access
The reimbursement criteria for Empaveli in Korea specify its use in adult PNH patients who either have contraindications to C5 inhibitors or have been treated with C5 inhibitors for over three months but have hemoglobin levels below 10.5 g/dL or require a switch due to side effects. This ensures that Empaveli is accessible to patients who need it most, without requiring prior review, unlike existing C5 inhibitors. This streamlined access is expected to improve the quality of life for PNH patients in Korea by providing a more effective and targeted treatment option.
