11 Dec Hemophilia: New Fusion Protein Allows For Less Frequent Dosing To Prevent Bleeding
MedicalResearch.com Interview with:
Dr. Elena Santagostino, MD PhD
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center
Ca’ Granda Foundation
Maggiore Hospital Policlinico, Milan, Italy
Medical Research: What is the background for this study? What are the main findings?
Dr. Santagostino: Two of our abstracts presented at the 57th ASH Annual Meeting are part of the PROLONG-9FP clinical program evaluating the efficacy and safety of CSL Behring’s investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). The first is an oral presentation on results from two Phase III studies and an ongoing extension study of rIX-FP for routine prophylaxis in previously-treated people with hemophilia B.
The two Phase III studies included adolescents and adults (ages 12 to 61) treated with dosing intervals of up to 14 days for 12 to 18 months, and children (ages 1 to 11) who received seven-day prophylaxis treatment for approximately 12 months. Overall, the median annualized spontaneous bleeding rate (AsBR) was 0.00 for all treatment intervals. The extension study is examining longer treatment intervals, including 10- and 14-day intervals in patients younger than 12 and more prolonged treatment intervals in patients older than 18, and so far has reported favorable long-term tolerability with no serious adverse reactions such as the development of inhibitors to factor IX or antibodies to rIX-FP.
A second abstract reported on a surgical sub-study in these trials found that a single pre-operative dose of rIX-FP maintained hemostasis during surgery with responses rated by investigators as “excellent” or “good.” Oven a 14-day perioperative period, patients needed six or seven infusions, and none developed inhibitors to factor IX or antibodies to rIX-FP.
Medical Research: What should clinicians and patients take away from your report?
Dr. Santagostino: These latest results are promising and confirm the data we saw in our pivotal studies, with prolonged dosing intervals of up to 14 days with rIX-FP for routine prophylaxis. This is an important attribute for patients who require a prophylactic regimen but don’t want treatment to disrupt their active lives.
Medical Research: What recommendations do you have for future research as a result of this study?
Dr. Santagostino: rIX-FP has been developed with a pharmacokinetic profile that allows for less frequent dosing. Through the ongoing extension study, we are continuing to evaluate longer treatment intervals and the potential impact on preventing bleeding in select patients.
Efficacy and safety of rIX-FP in previously-treated children, adolescents, and adults and
rIX-FP in previously-treated patients undergoing surgery
548 Efficacy and Safety Results of Prolong-9FP Clinical Program of Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) in Previously Treated Patients with Hemophilia B
Disorders of Coagulation or Fibrinolysis
Session: 322. Disorders of Coagulation or Fibrinolysis: Novel Treatment Strategies in Hemophilia Monday, December 7, 2015: 10:45 AM
W311ABCD, Level 3 (Orange County Convention Center)
Elena Santagostino, MD, PhD1, Christine Voigt, PhD2*, Denise Wolko2*, Grace Cole2*, Yanyan Li, PhD2* and Iris C. Jacobs, MD2
1Centro Emofilia e Trombosi, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy
2CRD, CSL Behring, King of Prussia, PA
2304 Successful Use of rFVIII, Turoctocog Alfa, during Orthopedic and Nonorthopedic Surgery in Patients with Severe Hemophilia A
Disorders of Coagulation or Fibrinolysis
Session: 322. Disorders of Coagulation or Fibrinolysis: Poster II
Sunday, December 6, 2015, 6:00 PM-8:00 PM
Hall A, Level 2 (Orange County Convention Center)
Luigi Solimeno, MD1*, Steven R. Lentz, MD, PhD2, Irina Matytsina, MD3*, Niels Zeuthen4*, Faraizah Abdul Karim, MD5*and Elena Santagostino, MD, PhD6
1Department of Orthopedic Surgery and Traumatology, Ca’ Grande Foundation, Maggiore Hosptial Policlinico, Milan, Italy
2University of Iowa Carver College of Medicine, Iowa City, IA
3Novo Nordisk A/S, Soeborg, Denmark
4Novo Nordisk A/S, Søborg, Denmark
5National Blood Center, Kuala Lumpur, Malaysia
6Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Ca’ Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy
Dr. Elena Santagostino, MD PhD (2015). Hemophilia: New Fusion Protein Allows For Less Frequent Dosing To Prevent Bleeding MedicalResearch.com