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Therapeutic Goods Administration Approves IDELVION® - CSL Behring’s Novel Recombinant Haemophilia B Treatment with up to 14-day Dosing Intervals

MELBOURNE, Australia — 03 October 2016

  • IDELVION is a long-acting recombinant albumin fusion protein for children and adults with haemophilia B, and is the first CSL Behring recombinant factor product to receive TGA approval
  • In clinical trials, IDELVION maintained factor IX activity levels above 5 percent over 14 days in the majority of patients undergoing routine prophylaxis, with a corresponding median annualized spontaneous bleeding rate (AsBR) of zero

CSL Behring announced that the Australian Therapeutic Goods Administration (TGA) has approved IDELVION® [albutrepenonacog alfa] in patients with haemophilia B (congenital factor IX deficiency).

IDELVION® is indicated in all patients with haemophilia B for:

  • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
  • Control and prevention of bleeding episodes
  • Control and prevention of bleeding in the perioperative setting

IDELVION is an innovative, long-acting albumin fusion protein linking recombinant coagulation factor IX with recombinant albumin and is appropriate for use in all age groups (children and adults).

IDELVION is the first and only Australian registered factor IX therapy that delivers high-level protection with up to 14-day dosing for appropriate patients. It helps patients maintain factor IX activity levels over a long period of time, facilitating greater patient freedom from frequent infusions. For patients receiving twice weekly factor IX, a switch to extended dosing intervals with IDELVION reduces the monthly factor IX consumption needed for prophylaxis therapy without comprising control of bleeding.

IDELVION provides excellent bleeding control by maintaining factor IX activity levels above 5 percent over a prolonged period of time,” said Loretta Croker, General Manager, CSL Behring Australia/New Zealand. “IDELVION delivers on CSL’s 100 year promise to develop and provide innovative specialty biotherapies that patients need and want. We are committed to bringing IDELVION to Australian patients with haemophilia B and particularly excited about the positive impact this long-acting therapy can have on their lives,” she said.

“The Australian Haemophilia Centre Director’s Organisation (AHCDO) views the development of effective long-acting clotting factor concentrates as a major step forward in the management of our patients with haemophilia,” said Dr Simon McRae, Consultant Haematologist and Chairman of AHCDO. “The ability to maintain clotting factor levels above a level that prevent the vast majority of bleeding events with less frequent infusions has the potential to improve long-term outcomes in individuals with haemophilia.”

The approval of IDELVION is based on results from the PROLONG-9FP clinical development program. PROLONG-9FP includes Phase I through Phase III open-label, multicenter studies evaluating the safety and efficacy of IDELVION in children and adults (ages 1 to 61 years) with haemophilia B (factor IX levels ≤ 2%).

“I have seen first-hand the benefits IDELVION has had on children with haemophilia B,” said Dr Julie Curtin, Senior Staff Specialist in Haematology, The Children’s Hospital at Westmead and an investigator in the PROLONG-9FP clinical development program. “IDELVION has enabled children on regular treatment with factor IX to reduce the frequency of infusions without increasing their risk of bleeding. For a child to only need an injection every 1-2 weeks is a good step forward in the management of haemophilia B which I welcome and I am sure my patients will welcome this improvement too.”

Gavin Finkelstein, President of the Haemophilia Foundation Australia said, “The community is very pleased to see the approval of new treatment opportunities for people with haemophilia B.”

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. In Australia, there are 530 people with haemophilia B (97 with severe haemophilia B) according to the 2014/2015 Australian Bleeding Disorders Registry (ABDR), a clinical registry for Australian patients with bleeding disorders.

About PROLONG-9FP
The data from the PROLONG-9FP pivotal Phase II/III clinical studies conducted in adults and adolescents showed median annualized spontaneous bleeding rates (AsBRs) of zero and factor IX activity levels above 5 percent in the majority of patients using IDELVION for routine prophylaxis. According to the World Federation of Hemophilia, patients with factor IX activity levels above 5 percent (and below 50 percent) are considered to have mild haemophilia. This result was achieved for both 14-day and 7-day dosing. The data for on-demand therapy showed that 93 percent of bleeds were controlled with one infusion, while 99 percent were controlled with one or two infusions. The most common adverse reactions observed in clinical trials were injection site reaction and headache. Results of the PROLONG-9FP studies conducted in children were generally consistent with those reported for adults and adolescents. Data from PROLONG-9FP were published in the American Society of Hematology’s publication Blood.

About IDELVION®
IDELVION, albutrepenonacog alfa is indicated in children and adults with haemophilia B for:

  • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
  • Control and prevention of bleeding episodes
  • Control and prevention of bleeding in the perioperative setting

IDELVION is not indicated for induction of immune tolerance in patients with haemophilia B.

IDELVION is contraindicated in patients who have a known hypersensitivity to the product, any of its components, excipients, or hamster protein.

CSL Behring engineered IDELVION to extend the half-life of recombinant factor IX through fusion with recombinant albumin. CSL Behring selected recombinant albumin as its fusion partner for its coagulation factor proteins due to its long physiological half-life. Additionally, recombinant albumin has been shown to have a good tolerability profile, low potential for immunogenic reactions and a well-known mechanism of clearance.

IDELVION is also approved in the European Union, United States, Switzerland, Japan and Canada.

About CSL Behring
CSL Behring is a global biotherapeutics leader which is driven by its promise to save lives. Focused on serving 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 more than 16,000 people with operations in more than 30 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring.

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Contact - Australia:
Jemimah Pentland
CSL Limited
Mobile: +61 412-635-483
Email: Jemimah.Pentland@csl.com.au

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

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