Non‐Inferiority of Subcutaneous Efepoetin Alfa Compared to Methoxy Polyethylene Glycol‐Epoetin Beta in Stage 3 or 4 CKD Patients: Insights From a Phase 3 Trial

• Published in: Nephrology (Carlton, Vic.) Vol. 30; no. 5; pp. e70046 - n/a
• Main Authors: Roger, Simon D., Wong, Chew Ming, Vejakama, Phisitt, Rahardjo, Kuspudji Dwitanto, Hsu, Bang‐Gee, Lee, Chang Meng, Wu, I‐Wen, Lee, Chin‐Chan, Situmorang, Tunggul Diapari, Krisanapan, Pajaree, Mazlan, Sadanah Aqashiah, Peng, Yu‐Sen, Sarwono, Johanes, De Asis, Norman, Solimen, Domingo, Jonny, Sangthawan, Pornpen, Chen, Jin‐Bor, Wang, Chia‐Liang, Chung, Sungjin, Villaflor, Agnes Jeans, Yang, Chul Woo, Kan, Wei‐Chih, Yang, Yu, Rubio‐Bicol, Jenny, Yan, Lee Yee, Lee, Sang Ho, Chiu, Yi‐Wen, Chen, Cheng‐Hsu, Na, Ki Young, Hassah, Wan Hasnul Halimi Wan, Kang, Young Sun, Choi, Bum‐Soon, Aquitania, Grace, Na, Ki Ryang, Wu, Mai‐Szu, Ahmad, Mohd Kamil, Isidto, Rey, Wu, Vincent, Leong, Goh Bak, Sung, Junne‐Ming, Noppakun, Kajohnsak, Chou, Kang‐Ju, Abdul Wahab, Mohamad Zaimi, Shin, Seok Joon, Nugroho, Pringgodigdo
• Format: Journal Article
• Language: English
• Published: Melbourne John Wiley & Sons Australia, Ltd 01.05.2025; Wiley Subscription Services, Inc
• Subjects: Adult; Aged; anaemia; Anemia; Anemia - blood; Anemia - diagnosis; Anemia - drug therapy; Anemia - etiology; Biomarkers - blood; chronic kidney disease; efepoetin alfa; Erythropoiesis; Erythropoietin; Erythropoietin - administration & dosage; Erythropoietin - adverse effects; Fc receptors; Female; Fusion protein; Hematinics - administration & dosage; Hematinics - adverse effects; Hemoglobin; Hemoglobins - metabolism; Humans; Injections, Subcutaneous; Kidney diseases; long‐acting erythropoiesis‐stimulating agent; Male; Middle Aged; Original; Polyethylene glycol; Polyethylene Glycols - administration & dosage; Polyethylene Glycols - adverse effects; Recombinant Fusion Proteins - administration & dosage; Recombinant Fusion Proteins - adverse effects; Recombinant Proteins; Renal Insufficiency, Chronic - blood; Renal Insufficiency, Chronic - complications; Renal Insufficiency, Chronic - diagnosis; Response rates; Time Factors; Treatment Outcome; chronic kidney disease; efepoetin alfa; anaemia; long‐acting erythropoiesis‐stimulating agent
• ISSN: 1320-5358; 1440-1797; 1440-1797
• DOI: 10.1111/nep.70046

ABSTRACT Aim Efepoetin alfa, a novel long‐acting erythropoietin (EPO)‐hybrid Fc fusion protein, represents a promising erythropoiesis‐stimulating agent (ESA) for addressing anaemia in chronic kidney disease (CKD) patients. This Phase 3 trial was to assess the efficacy and tolerability of subcutaneous efepoetin alfa in comparison to subcutaneous methoxy polyethylene glycol‐epoetin beta in stage 3 or 4 CKD patients. Methods A randomised, multicentre, open‐label Phase 3 trial enrolled 391 CKD stage 3 or stage 4 patients. Subjects underwent a 20‐week correction period followed by an 8‐week evaluation period. Responders continued treatment for an extra 24‐week extension to evaluate long‐term safety, maintenance effectiveness, and the longer treatment interval. Results In the efepoetin alfa Q2W (every 2 weeks) group, the response rate was 75.6%; while in the methoxy polyethylene glycol‐epoetin beta Q2W group, the response rate was 69.3%. The difference in the response rate was 6.3% with 95% CI (confidence interval) –3.1% to 15.5%. The lower limit of the 95% CI was above the prespecified non‐inferiority margin of −9.0%. Adverse event rates were comparable between the treatment groups. Conclusion Efepoetin alfa demonstrated non‐inferiority to methoxy polyethylene glycol‐epoetin beta in correcting anaemia and maintaining haemoglobin (Hb) levels among stage 3 and 4 CKD patients. Moreover, the safety profile of efepoetin alfa was comparable to methoxy polyethylene glycol‐epoetin beta.