rIX-FP Subct / CSL Behring - LARVOL DELTA

Safety, Efficacy and Treatment Patterns of rIX-FP in Previously Untreated Paediatric Haemophilia B Patients: A Retrospective Chart Review in Japan. (PubMed, Haemophilia) - "This study provides real-world insights into rIX-FP use in paediatric PUPs, demonstrating its favourable safety, effective prophylaxis, and lower IVR in infants."

Journal • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia B • Pediatrics • Rare Diseases

Characterization of recombinant Factor IX fusion proteins enabling subcutaneous administration. (PubMed, J Thromb Haemost) - "rIX(R338L)-FP was shown to be a promising candidate for SC administration, exhibiting increased specific activity combined with higher activity-based exposure, and indicating efficacy at lower protein dose."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B (clinicaltrials.gov) - P3b; N=95; Completed; Sponsor: CSL Behring; Active, not recruiting ➔ Completed

Clinical • Trial completion • Hematological Disorders • Hemophilia • Rare Diseases

[VIRTUAL] ANALYSIS OF EXTENDED DOSING REGIMENS WITH RECOMBINANT COAGULATION FACTOR IX ALBUMIN FUSION PROTEIN (RIX‐FP) IN ADULT AND PAEDIATRIC PATIENTS WITH HAEMOPHILIA B: A POPULATION PHARMACOKINETIC SIMULATION (EAHAD 2021) - "FIX activity-time profiles for different extended dosing intervals can be predicted using the PPK model. The PPK simulation data suggest that most adult and paediatric patients with haemophilia B receiving rIX-FP on 21-day and 14-day extended interval dosing regimens, respectively, could reach adequate FIX activity levels associated with a mild or moderate haemophilia classification as seen in the clinical trial."

Clinical • PK/PD data • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases