KING OF PRUSSIA, Pa., June 20, 2016 /PRNewswire/ -- Driven by its promise to save lives, CSL Behring announced today that AFSTYLA® [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor VIII single-chain therapy for adults and children with hemophilia A, is now available nationwide. AFSTYLA is the first and only single-chain product for hemophilia A specifically designed for long-lasting protection from bleeds with two to three times weekly dosing.
AFSTYLA was approved by the U.S. Food and Drug Administration (FDA) in May for use in children and adults 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. 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.
"We are proud to add AFSTYLA to our leading portfolio of bleeding disorder products and are particularly excited about the positive impact that treatment with AFSTYLA can have on the lives of patients with hemophilia A," said Jerry Powell, M.D., Medical Director, North America Commercial Operations, CSL Behring. "AFSTYLA is the second product from our innovative recombinant factor development program approved by the FDA in 2016. In March, IDELVION® [Coagulation Factor IX (Recombinant), Albumin Fusion Protein], the first and only recombinant albumin fusion protein for hemophilia B, was approved by the FDA. Both AFSTYLA and IDELVION deliver on our promise to develop and provide innovative specialty biotherapies that help people with serious medical conditions live full lives."
AFSTYLA is available in five vial sizes: 250, 500, 1000, 2000 and 3000 international units of factor VIII. The product can be stored in the refrigerator or at room temperature (2-25°C; 36-77°F).
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.
FDA approval of AFSTYLA was based on results from the AFFINITY clinical development program. AFFINITY included 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.
Data from the AFFINITY clinical development program show a median annualized spontaneous bleeding rate (AsBR) of 0.00 in prophylactic use for both the adult and adolescent study and 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. 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 16 total surgical procedures (involving 13 adult or adolescent patients in the study), the 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.
AFSTYLA (also known as rVIII-Single Chain) for hemophilia A is CSL Behring's recombinant single-chain factor VIII specifically designed for increased molecular stability and long 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.
AFSTYLA 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.
IDELVION Important Safety Information
IDELVION®, Coagulation Factor IX (Recombinant), Albumin Fusion Protein (rIX-FP), is indicated in children and adults with hemophilia B (congenital factor IX deficiency) for:
- On-demand control and prevention of bleeding episodes
- Perioperative management of bleeding
- Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
IDELVION is not indicated for induction of immune tolerance in patients with hemophilia B.
IDELVION is contraindicated in patients who have had life-threatening hypersensitivity to the product or its components, including hamster proteins.
IDELVION is for intravenous use only. IDELVION 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 or more frequent dosing may be needed for pediatric patients.
Hypersensitivity reactions, including anaphylaxis, are possible. Advise patients who self-administer to immediately report symptoms of hypersensitivity, including angioedema, chest tightness, hypotension, generalized urticaria, wheezing, and dyspnea. If symptoms occur, discontinue IDELVION and administer appropriate treatment.
Development of neutralizing antibodies (inhibitors) to IDELVION may occur. If expected factor IX activity plasma levels are not attained or bleeding is not controlled with appropriate dose, perform an assay to measure factor IX inhibitor concentration. Factor IX activity assay results may vary with the type of activated partial thromboplastin time reagent used.
Thromboembolism (e.g., pulmonary embolism, venous thrombosis, and arterial thrombosis) can occur when using factor IX-containing products. In addition, nephrotic syndrome has been reported following immune tolerance induction in hemophilia B patients with factor IX inhibitors and allergic reactions to factor IX.
The most common adverse reaction (incidence ≥1%) reported in clinical trials was headache.
Please see full prescribing information for IDELVION, including patient product information.
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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