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New Data Demonstrate Prophylactic Treatment with IDELVION® Reduces Bleed Frequency and Has Potential to Positively Impact Patients with Haemophilia B

Findings presented at the International Society on Thrombosis and Haemostasis Congress 2017 show product’s impact on treatment adherence, consumption and quality of life

BERLIN — 12 July 2017

CSL Behring today presented data finding that prophylaxis treatment with IDELVION® provides consistently high factor IX levels, resulting in low bleeding rates in both adult and paediatric patients with haemophilia B. Results were presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress 2017 in Berlin. IDELVION, CSL Behring’s novel, long-acting albumin fusion protein linking recombinant coagulation factor IX (FIX) with recombinant albumin for the treatment of haemophilia B, is the first rIX-FP therapy that delivers high-level protection with up to 14-day dosing in appropriate patients.

“When treating patients with haemophilia B, our goal is to reduce bleed frequency by maintaining high FIX activity trough levels,” said Giancarlo Castaman, MD, Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital. “Our findings add to the growing body of evidence demonstrating the efficacy of IDELVION and the product’s potential to positively impact patients by offering high and sustained factor levels that provide persistent bleed prevention.”

Additional data presented at the Congress show that IDELVION prophylaxis regimens result in high rates of compliance, reduced consumption compared with previous FIX in both adult and paediatric patients, and improvement in paediatric health-related quality of life (HRQoL).

Key Study Findings

High and sustained observed trough FIX activity levels with prophylactic dosing of IDELVION in patients with haemophilia B (Poster #1772)

  • Analysis evaluated trough FIX activity levels in paediatric and adult patients with haemophilia B during prophylaxis with IDELVION.
  • A total of 45 and 18 patients at least 12 years of age with haemophilia B were treated with IDELVION prophylactically once every 7 and 14 days (mean doses 47 and 74 IU/kg), respectively. Paediatric patients younger than 12 years of age (N=24) received prophylaxis once every 7 days (mean dose 47 IU/kg).
  • In adult/adolescent patients, once every 7- and 14-day prophylaxis dosing regimens provided mean observed trough FIX levels of 22.4 percent and 12.7 percent, respectively. Paediatric patients had mean FIX activity levels of 12.4 percent.

High adherence in adult and paediatric patients with haemophilia B receiving prophylaxis with rIX-FP (Poster #1816)

  • Study evaluated adherence to various IDELVION treatment regimens among adult and paediatric patients with haemophilia B.
  • Adults with haemophilia B received either on-demand treatment for 6 months then prophylaxis every 7 days (on-demand arm; n=23) or 7-day prophylaxis for 6 months then, if eligible, prophylaxis once every 10 or 14 days (prophylaxis arm; n=40). Paediatric patients (n=27) received prophylaxis every 7 days.
  • In the adult study, 94.9 percent of subjects were adherent to their prophylaxis schedule. Mean prophylaxis compliance rate for the 7-day regimen was similar between initial on-demand and prophylaxis arms (95.5 percent and 94.7 percent, respectively). Mean compliance rates with 10- and 14-day regimens were 90.7 percent and 97.2 percent, respectively. Overall, 85.7 percent of adult patients were dose compliant (within 10 percent of prescribed dose at least 80 percent of the time).
  • In the paediatric study, all patients were adherent with the weekly prophylaxis schedule; mean overall compliance rate was 97.9 percent and was similar between those aged 1–5 years and those aged 6–11 years.

Prophylaxis with rIX-FP reduces consumption compared with previous FIX in both adult and paediatric patients (Poster #952)

  • The impact of an extended dosing interval on FIX consumption was assessed in adult and paediatric patients with haemophilia B enrolled in two clinical trials.
  • Adults and adolescents were treated prophylactically every 7, 10 or 14 days and paediatric patients were treated prophylactically every 7 days. Consumption of FIX before and during study was compared.
  • Compared to previous treatment, prophylaxis with the prolonged dose interval afforded by IDELVION resulted in a substantial decrease in total FIX consumption and a subsequent reduction in the burden of treatment in this group of patients.

Health-related quality of life in paediatric haemophilia B patients treated with rIX-FP (Poster #1105)

  • Study evaluated the impact of IDELVION prophylaxis on HRQoL in paediatric patients with haemophilia B.
  • The impact of rIX-FP prophylaxis on HRQoL was assessed in paediatric patients (4–7 years [n=12], 8–12 years [n=7]) receiving weekly prophylaxis with 35–50 IU/kg rIX-FP as part of a multicentre Phase III study.
  • Children showed overall HRQoL improvement between baseline and end of study. Parents reported an improvement in treatment satisfaction.

About Haemophilia B

Haemophilia B is a congenital bleeding disorder characterized by deficient or defective factor IX; nearly all affected patients are male. People with haemophilia B may experience prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. According to the U.S. Centers for Disease Control and Prevention, the condition affects approximately one in 25,000 male births.

About CSL Behring

CSL Behring is a global biotherapeutics leader which is driven by its promise to serve patients’ needs by using the latest technologies. We develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL), headquartered in Melbourne, Australia, employs nearly 20,000 people, delivering its life-saving therapies to people in more than 60 countries. For more information visit www.CSLBehring.com and follow us on www.Twitter.com/CSLBehring.

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Contact:
Greg Healy
CSL Behring
Office: +1-610-878-4841
Mobile: +1-610-906-4564
Email: Greg.Healy@CSLBehring.com

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