Therapeutic Goods Administration Approves AFSTYLA® - CSL Behring's Novel Recombinant Haemophilia A Treatment

- AFSTYLA is the first and only single-chain recombinant factor VIII specifically designed to treat haemophilia A

-AFSTYLA reduces the number of injections required for prophylaxis therapy with twice or three times a week dosing, without compromising control of bleeding

- In clinical trials, patients undergoing prophylaxis with AFSTYLA experienced a median annualised spontaneous bleeding rate (AsBR) of zero

- Real-world AFSTYLA trial design demonstrated effective bleed prevention with reduced dosing frequency can be achieved with low FVIII consumption

- AFSTYLA was well tolerated, with no patients discontinuing the study due to adverse events

News provided by

CSL Behring

Apr 19, 2017, 14:00 ET

MELBOURNE, Australia, April 19, 2017 /PRNewswire/ -- Global biotherapeutics leader, CSL Behring today announced that the Australian Therapeutic Goods Administration (TGA) has approved AFSTYLA^® [lonoctocog alfa] a recombinant single chain coagulation factor VIII (rFVIIISingleChain) in patients with haemophilia A (congenital factor VIII deficiency).

AFSTYLA is indicated in all patients with haemophilia A for:

Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
Control and prevention of bleeding episodes
Perioperative management (surgical prophylaxis)

AFSTYLA is the first and only single-chain recombinant factor VIII product to treat haemophilia A. The single chain design provides sustained protection from bleeds with a strong binding affinity to Von Willebrand factor. In clinical trials, patients undergoing prophylaxis with AFSTYLA experienced a median annualised spontaneous bleeding rate (AsBR) of zero with two to three times weekly dosing. AFSTYLA was demonstrated to be well tolerated with no patients discontinuing the study due to adverse events.

For an estimated 81% of severe haemophilia A patients who receive prophylaxis every second day or three times a week (McRae S, 2016), the switch to extended dosing intervals with AFSTYLA will reduce the number of injections required for prophylaxis therapy without compromising control of bleeding.

Dr. Andrew Cuthbertson, Chief Scientific Officer and Director R&D, CSL Limited said, "TGA approval of AFSTYLA - an innovative and effective haemophilia A therapy - further demonstrates CSL Behring's dedication and century long promise to develop and deliver novel treatments that have the potential to improve patients' lives. We are very excited to add this treatment to our industry-leading portfolio of coagulation therapies."

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

Approval of AFSTYLA is based on results from the AFFINITY clinical development program. AFFINITY includes Phase I through to Phase III open-label, multicentre studies evaluating the safety and efficacy of AFSTYLA in children and adults (ages 1 to 61 years) with severe haemophilia A.

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 A."

CSL Behring is committed to bringing AFSTYLA to Australian patients and intends to work with the health care community to provide effective treatment options for people with haemophilia A.

About Haemophilia A
Haemophilia A is a congenital bleeding disorder characterised by deficient or defective factor VIII; nearly all affected patients are male. People with Haemophilia A may experience prolonged or spontaneous bleeding, especially into the muscles and joints. In Australia, there are 2158 people with Haemophilia A (640 with severe Haemophilia A) according to the Australian Bleeding Disorders Registry (NBA, 2015).

About AFFINITY
AFFINITY includes two pivotal and one extension open-label multi-centre study evaluating the safety and efficacy of AFSTYLA in children, adolescents and adults with haemophilia A. Data from AFFINITY were recently published in the American Society of Hematology's publication Blood and in the Journal of Thrombosis and Haemostasis. Data comparing the pharmacokinetics of AFSTYLA and octocog alfa in patients with severe haemophilia A were recently published in The World Federation of Hemophilia's journal Haemophilia.

About AFSTYLA^®AFSTYLA (rVIII-SingleChain) for haemophilia A is CSL Behring's recombinant single-chain factor VIII specifically designed for greater molecular stability and longer duration of action. AFSTYLA uses a covalent bond to form one structural entity, a single polypeptide-chain, to improve the stability of factor VIII and provide factor VIII activity with the option of twice weekly dosing. The single-chain Factor VIII molecule of AFSTYLA was originated by SK Chemicals Co. Ltd., a bio-pharmaceutical company in Korea, and licensed to CSL Behring in 2009.

Safety and Tolerability of AFSTYLA
AFSTYLA was demonstrated to be well tolerated with no patients discontinuing the study due to adverse events. Hypersensitivity, dizziness, paraesthesia, rash, pyrexia and injection site reactions were the most common adverse events reported. One event of hypersensitivity was reported but the patient continued in the study. No inhibitors have been observed in previously treated patients. CSL Behring is currently conducting an extension study that includes previously untreated patients (PUPs), to determine the safety, efficacy and incidence of inhibitors in this high risk population. AFSTYLA is contraindicated in patients who have had life-threatening hypersensitivity reactions, including anaphylaxis to AFSTYLA, any of its components, or hamster proteins. Precautions for use include hypersensitivity reactions and inhibitors. If available, the chromogenic assay should be used to determine FVIII activity.

AFSTYLA^® is also approved in the European Union, United States, and Canada.

About CSL Behring

CSL Behring is a global biotherapeutics leader 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 17,000 people, providing its life-saving, life-changing therapies to people in more than 60 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring.

SOURCE CSL Behring