Japan's Ministry of Health, Labour and Welfare 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, previously treated patients undergoing prophylaxis with AFSTYLA® experienced a median annualised spontaneous bleeding rate (AsBR) of zero

- Real-world AFSTYLA® trial design demonstrated that effective bleed prevention with reduced dosing frequency can be achieved with low factor VIII consumption

-AFSTYLA® was well tolerated, with no patients discontinuing the study due to adverse drug reaction

News provided by

CSL Behring

Sep 28, 2017, 10:30 ET

TOKYO, Sept. 28, 2017 /PRNewswire/ -- Global biotherapeutics leader CSL Behring today announced that Japan's Ministry of Health, Labor and Welfare (MHLW) has approved AFSTYLA^® (hereafter AFSTYLA) [lonoctocog alfa] a recombinant single-chain coagulation factor VIII (rVIII-SingleChain) 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
On-demand treatment and control of bleeding
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 due to the greater molecular stability and duration of action through a strong affinity with von Willebrand factor (vWF). 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 drug reactions.

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 0 to 65 years) with severe haemophilia A.

CSL Behring is committed to bringing AFSTYLA to Japanese 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; most patients are male. People with Haemophilia A may experience prolonged or spontaneous bleeding, especially into the muscles and joints.

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 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.
*: NDA in Japan was submitted based on the completed two pivotal studies for review

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 drug reaction. Hypersensitivity, dizziness, paraesthesia, rash, pyrexia and injection site reactions were the most common adverse drug reaction 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.

AFSTYLA is also approved in the European Union, United States, Canada, Switzerland and Australia.

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 haemolytic 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.co.jp.

SOURCE CSL Behring