A Randomized, Two-Armed, Double-Blind, Single-Dose, Cross-Over, Bioequivalence Clinical Trial to Compare Pharmacokinetic Parameters and Safety of Recombinant Human Factor Viii With Fc Fusion Produced by Aryogen Pharmed Company Versus Elocta® (Reference Product) in Previously Treated Patients With Severe Haemophilia A Publisher

Summary: A study confirms AryoGen’s rFVIII-Fc is bioequivalent to Elocta®, offering a new treatment for haemophilia A patients. #Haemophilia #Biotech

Eghbali A¹ ; Eshghi P² ; Toogeh G³ ; Alavi S² ; Badiei Z⁴ ; Ghanavat M⁵ ; Bordbar M⁶ ; Bazrafshan A⁷ ; Karimi K³ ; Ahmadinejad M⁸ ; Sabzvari A⁹ ; Kafi H¹⁰
Source: Annals of Hematology Published:2025

Abstract

This clinical study evaluates the bioequivalence of recombinant factor VIII with Fc fusion protein (rFVIII-Fc) developed by AryoGen Pharmed Company compared to the reference product, Elocta® by Sobi Co., in severe haemophilia A patients. Fc-fused recombinant factor VIII represents a significant advancement in haemophilia A treatment, offering extended half-life and reduced infusion frequency, thus improving patients’ adherence to treatment and quality of life. In a randomized, double-blind, single-dose crossover trial, 50 Iranian patients were assigned to treatment groups in a 1:1 ratio. Subjects received both the test and the reference product with a 7-day washout period between treatments. Pharmacokinetic assessments were conducted over five days post-administration to evaluate the primary outcome, the dose-normalized area under the curve (DNAUC). The results established bioequivalence between rFVIII-Fc (AryoGen Pharmed Company) and Elocta®, based on the DNAUC as the primary outcome, in which the ratio of test and reference products was calculated to be 108.56 (90% confidence interval 104.88 to 112.37), falling within the pre-defined equivalence margin of 80–125%. Secondary outcomes, including area under the curve (AUCinf), maximum concentration (Cmax), and half-life, further supported bioequivalence. Safety profiles were comparable, with adverse events mainly related to haemophilia A rather than the intervention. In conclusion, the rFVIII-Fc product is bioequivalent to Elocta® with a similar safety profile, offering an effective alternative for severe haemophilia A patients. This trial was registered in ClinicalTrials.gov (NCT06137092). © The Author(s) 2025.