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U.S. FDA Approves CSL Behring’s AFSTYLA®-- The First and Only Recombinant Factor VIII Single Chain Therapy for Hemophilia A

KING OF PRUSSIA, Pa. — 26 May 2016

  • AFSTYLA is the first and only single-chain product for hemophilia A, specifically designed for long-lasting protection from bleeds with twice-weekly dosing available
  • In clinical trials, patients undergoing prophylaxis with AFSTYLA experienced a median annualized spontaneous bleeding rate (AsBR) of 0.00
  • AFSTYLA demonstrated a strong safety profile with no inhibitors observed in clinical trials

CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved AFSTYLA® [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor VIII single-chain therapy for adults and children with hemophilia A. AFSTYLA is the first and only single-chain product for hemophilia A that is specifically designed for long-lasting protection from bleeds with two to three times weekly dosing. In clinical trials, patients undergoing prophylaxis with AFSTYLA experienced a median annualized spontaneous bleeding rate (AsBR) of 0.00. Once activated, AFSTYLA is identical to natural factor VIII. Clinical trials of AFSTYLA demonstrated a strong safety profile with no inhibitors observed.

AFSTYLA is indicated in adults and children with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes; on-demand treatment and control of bleeding episodes; and the perioperative management of bleeding. AFSTYLA is expected to be available early this summer.

“FDA’s approval of the first recombinant single-chain therapy that offers long-lasting hemostatic efficacy provides an important new treatment option for patients and healthcare providers as it has been specifically designed for increased molecular stability and duration of action,” said Dr. Lisa Boggio, Assistant Professor of Internal Medicine, Hematology and Oncology, Clinical Director of the Rush Hemophilia and Thrombophilia Center, and AFFINITY clinical development program study investigator. “AFSTYLA offers patients an opportunity for excellent efficacy with a strong safety profile and twice-weekly dosing -- potentially helping patients to fit treatment into their active lives.”

The approval of AFSTYLA is based on results from the AFFINITY clinical development program. AFFINITY includes two pivotal and one extension open-label multi-center studies evaluating the safety and efficacy of AFSTYLA in children, adolescents and adults with hemophilia A.

"For 100 years, CSL has focused on researching and developing innovative therapies that meet the treatment challenges patients face,” said Dr. Andrew Cuthbertson, Chief Scientific Officer and Director of R&D, CSL Limited. "The approval of AFSTYLA, an innovative and effective hemophilia A therapy, further demonstrates CSL’s dedication to developing and delivering novel therapies that have the potential to improve patients’ lives. We are very excited to add this treatment to our industry-leading portfolio of coagulation therapies and look forward to the positive impact AFSTYLA can have on patients with hemophilia A.”

About Hemophilia A

Primarily affecting males, hemophilia A is a congenital bleeding disorder characterized by deficient or defective factor VIII. People with hemophilia A may experience prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs. According to the United States Centers for Disease Control and Prevention (CDC), the condition affects approximately 1 in 6,000 male births.

About AFFINITY

The data from the AFFINITY clinical development program showed a median annualized spontaneous bleeding rate (AsBR) of 0.00 in both the adult and adolescent study as well as the pediatric study. The median annualized bleeding rate (ABR) was 1.14 in adult and adolescent patients and 3.69 in children less than 12 years of age using AFSTYLA prophylactically. Of 1,195 bleeds treated in the pivotal study (848 in adults and adolescents; 347 in children), 94 percent of bleeds in adult and adolescent patients and 96 percent of bleeding events in pediatric patients were effectively controlled with no more than two infusions of AFSTYLA weekly; 81 percent of bleeds in adult and adolescent patients and 86 percent of bleeding events in pediatric patients were effectively controlled by only one infusion. The majority of bleeding events treated with AFSTYLA (94 percent in adults and adolescents; 96 percent in children) were rated as excellent or good. Of the 13 adult or adolescent patients in the study who underwent surgical procedures (16 total surgeries), hemostatic efficacy of AFSTYLA was rated as excellent (15 times) or good (once). The most common adverse reactions reported in clinical trials were dizziness and hypersensitivity.

About AFSTYLA®

AFSTYLA (also known as rVIII-Single Chain) for hemophilia 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 that forms one structural entity, a single polypeptide-chain, to improve the stability of factor VIII and provide longer-lasting factor VIII activity. Regulatory agencies in Europe, Switzerland and Australia are currently reviewing CSL Behring’s license applications for AFSTYLA.

For more information about AFSTYLA, including full prescribing information, please visit http://labeling.cslbehring.com/PI/US/Afstyla/EN/Afstyla-Prescribing-Information.pdf.

Important Safety Information

AFSTYLA®, Antihemophilic Factor (Recombinant), Single Chain, is indicated in adults and children with hemophilia A (congenital Factor VIII deficiency) for:

  • On-demand treatment and control of bleeding episodes
  • Routine prophylaxis to reduce the frequency of bleeding episodes
  • Perioperative management of bleeding

AFSTYLA is not indicated for the treatment of von Willebrand disease.

AFSTYLA is contraindicated in patients who have had life-threatening hypersensitivity reactions to AFSTYLA or its excipients, or to hamster proteins.

AFSTYLA is for intravenous use only. AFSTYLA can be self-administered or administered by a caregiver with training and approval from a healthcare provider or hemophilia treatment center. Higher dose per kilogram body weight and/or more frequent dosing may be needed for patients under 12 years of age. Hypersensitivity reactions, including anaphylaxis, are possible. Advise patients to immediately report symptoms of a hypersensitivity reaction. If symptoms occur, discontinue AFSTYLA and administer appropriate treatment.

Development of factor VIII (FVIII) neutralizing antibodies (inhibitors) can occur. If expected factor VIII activity levels are not attained or bleeding is not controlled with appropriate dose, perform an assay to measure factor VIII inhibitor concentration.

Monitor plasma factor VIII activity using a chromogenic assay or one-stage clotting assay. If one-stage clotting assay is used, multiply result by a conversion factor of 2 to determine factor VIII activity level.

The most common adverse reactions (>0.5%) reported in clinical trials were dizziness and hypersensitivity.

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 about CSL Behring visit www.CSLBehring.com or follow us at www.Twitter.com/CSLBehring.

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Contact:

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

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