04 March 2016
U.S. FDA Approves CSL Behring’s IDELVION® -- The First and Only Hemophilia B Therapy with Up to 14-day Dosing Intervals
CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved IDELVION®
[Coagulation Factor IX (Recombinant), Albumin Fusion Protein], its novel, long-acting albumin fusion protein linking recombinant coagulation factor IX with recombinant albumin for the treatment of hemophilia B. IDELVION
is the first and only factor IX therapy that delivers high-level protection with up to 14-day dosing in appropriate patients. This dosing interval has been achieved while maintaining high levels of factor activity, above 5 percent over 14 days at 75 IU/kg. This reduces the monthly number of units needed for prophylaxis therapy.
23 February 2016
First Export of Privigen® From New Manufacturing Facility in Broadmeadows, Melbourne, Australia
Global biotherapeutics leader CSL Behring today announced an increase in the global supply of Privigen®
as the first export has been shipped from a new state-of-the-art manufacturing facility in Broadmeadows, Melbourne, Australia. The new Turner Privigen Facility, named for Mr. Peter J. Turner, former President of CSL Behring and former Chief Operating Officer of CSL group, will substantially increase CSL Behring’s total immunoglobulin (Ig) therapy production capabilities.
07 December 2015
CSL Behring Presents Phase III Data for Its Long-Acting Recombinant Factor IX Albumin Fusion Protein for Hemophilia B at the 57th ASH Annual Meeting & Exposition
Global biotherapeutics leader CSL Behring today presented data from its Phase III PROLONG-9FP clinical program evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP). The data, from an ongoing extension study and two pivotal Phase III studies, assessed rIX-FP for routine prophylaxis in previously-treated adults with hemophilia B, at dosing intervals of up to 14 days. The findings were presented during an oral presentation at the American Society of Hematology’s (ASH) 57th ASH Annual Meeting and Exposition in Orlando, along with a second abstract reporting efficacy and safety results of rIX-FP in patients undergoing surgical procedures.
02 December 2015
CSL Behring to Present New Data for rIX-FP and rVIII-SingleChain at the 57th ASH Annual Meeting & Exposition
CSL Behring announced today that it will present several abstracts from its recombinant coagulation factor development programs at the American Society of Hematology’s (ASH) 57th ASH Annual Meeting in Orlando, December 5-8. An oral presentation will focus on the pivotal trial for CSL Behring’s novel, long-acting albumin fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) for the treatment of hemophilia B.
08 September 2015
Centers for Medicare and Medicaid Services Extends New Technology Add-On Payment for CSL Behring’s Kcentra® for Third Year
CSL Behring today announced that the Centers for Medicare and Medicaid Services (CMS) again extended the new technology add-on payment (NTAP) for Kcentra®
(Prothrombin Complex Concentrate [Human]). The NTAP for Kcentra
is available through September 2016 for eligible Medicare beneficiaries treated in the inpatient hospital setting. Kcentra
is the first and only non-activated 4-factor prothrombin complex concentrate (4F-PCC) approved by the U.S. Food and Drug Administration (FDA) for the urgent reversal of acquired coagulation factor deficiency induced by Vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with acute major bleeding or in need of an urgent surgery or invasive procedure. Kcentra
, first approved for use in the U.S. in April 2013, received its NTAP designation effective October 1, 2013.
Important Safety Information for AFSTYLA
AFSTYLA®, Antihemophilic Factor (Recombinant), Single Chain, 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 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 FVIII activity levels are not attained or bleeding is not controlled with appropriate dose, perform an assay to measure FVIII inhibitor concentration.
Monitor plasma FVIII 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 FVIII activity level.
The most common adverse reactions reported in clinical trials (>0.5%) were dizziness and hypersensitivity.
Indications for AFSTYLA
AFSTYLA 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 frequency of bleeding episodes
- Perioperative management of bleeding
AFSTYLA is not indicated for the treatment of von Willebrand disease.
Please see full prescribing information for AFSTYLA.
Important Safety Information for IDELVION
IDELVION®, Coagulation Factor IX (Recombinant), Albumin Fusion Protein (rFIX-FP), 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 (eg, 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.
Indications for IDELVION
IDELVION 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.
Please see full prescribing information for IDELVION.
Important Safety Information for Berinert
Berinert®, C1 Esterase Inhibitor (Human), is a plasma-derived concentrate of C1 Esterase Inhibitor (Human), indicated for the treatment of acute abdominal, facial or laryngeal attacks of hereditary angioedema (HAE) in adult and adolescent patients. The safety and efficacy of Berinert for prophylactic therapy have not been established.
Berinert is contraindicated in individuals with a history of life-threatening systemic reactions to C1 esterase inhibitor preparations (including anaphylaxis).
Monitor patients for early signs of allergic or hypersensitivity reactions (including hives, generalized urticaria, chest tightness, wheezing, hypotension, and anaphylaxis). If hypersensitivity is suspected, immediately discontinue administration of Berinert and initiate appropriate treatment. Epinephrine should be immediately available for treatment of acute severe hypersensitivity reactions.
Serious arterial and venous thromboembolic events have been reported at recommended doses of C1 Esterase Inhibitor (Human) products, including Berinert, following administration to patients with HAE. Risk factors may include having an indwelling venous catheter/access device; prior history of thrombosis; underlying atherosclerosis; use of oral contraceptives or certain androgens; morbid obesity; and immobility. Weigh benefits/risks before administering to patients with known risk factors for TE events and closely monitor such patients during and after Berinert administration. TE events also have been reported with C1 Esterase Inhibitor (Human) products when used for unapproved indications at higher than recommended doses.
Patients able to recognize signs and symptoms of HAE attack and comprehend necessary training can self-administer Berinert. Patients should not attempt to self-administer unless they have been trained and determined to be capable by healthcare provider. Advise patients to seek medical attention immediately following self-administration for laryngeal attacks, and to seek medical attention if progress of any attack makes them unable to properly prepare or administer dose of Berinert.
Berinert is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.
The most serious adverse reaction reported in subjects who received Berinert in clinical studies was an increase in the severity of pain associated with HAE. Dysgeusia was the most common adverse reaction reported in over 4% of subjects and more frequently than in the placebo group.
Berinert has not been evaluated in pregnant women or nursing mothers, and should be used only if clearly needed. The safety and efficacy of Berinert have not been established in children (ages 0 through 12) or in the geriatric population. In clinical trials, the half-life of Berinert was shorter and clearance was faster in children than in adults; the clinical implication is not known.
Please see full prescribing information for Berinert, including the patient product information.
Important Safety Information for Privigen
Immune Globulin Intravenous (Human), 10% Liquid, Privigen®, is indicated as replacement therapy for patients with primary immunodeficiency (PI) associated with defects in humoral immunity, including but not limited to common variable immunodeficiency (CVID), X-linked agammaglobulinemia, congenital agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. Privigen is also indicated to raise platelet counts in patients with chronic immune thrombocytopenic purpura (ITP).
WARNING: THROMBOSIS, RENAL DYSFUNCTION AND ACUTE RENAL FAILURE
- Thrombosis may occur with immune globulin products, including Privigen. Risk factors may include advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors.
- Renal dysfunction, acute renal failure, osmotic nephrosis, and death may occur with the administration of human immune globulin intravenous (IGIV) products in predisposed patients. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products that contain sucrose. Privigen does not contain sucrose.
- For patients at risk of thrombosis, renal dysfunction or renal failure, administer Privigen at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity.
See full prescribing information for complete boxed warning.
Privigen is contraindicated in patients with history of anaphylactic or severe systemic reaction to human immune globulin, in patients with hyperprolinemia, and in IgA-deficient patients with antibodies to IgA, who have had hypersensitivity reactions. Patients with IgA deficiency and antibodies to IgA are at greater risk of severe hypersensitivity and anaphylactic reactions.
In patients at risk for developing acute renal failure, monitor urine output and renal function, including blood urea nitrogen and serum creatinine; discontinue if renal function deteriorates. Ensure that patients with preexisting renal insufficiency or otherwise predisposed are not volume-depleted and administer Privigen at the minimum rate of infusion practicable.
Thrombosis might occur with Privigen, even in the absence of known risk factors. Patients could also experience hyperproteinemia, increased serum viscosity, or hyponatremia; infrequently, aseptic meningitis syndrome (AMS) may occur—more frequently with high doses (2 g/kg) and/or rapid infusion.
Hemolysis, either intravascular or due to enhanced red blood cell sequestration, can develop subsequent to treatment. Risk factors include non-O blood group, underlying inflammation, and high doses. Closely monitor patients for hemolysis and hemolytic anemia. Consider the relative risks and benefits before prescribing high-dose regimen for chronic ITP in patients at increased risk of thrombosis, hemolysis, acute kidney injury or volume overload. Monitor patients for pulmonary adverse reactions and signs of transfusion-related acute lung injury (TRALI).
Privigen is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.
In clinical studies of patients being treated with Privigen for PI, the most common adverse reactions observed in >5% of subjects were headache, fatigue, nausea, chills, vomiting, back pain, pain, elevated body temperature, abdominal pain, diarrhea, cough, stomach discomfort, chest pain, joint swelling/effusion, influenza-like illness, pharyngolaryngeal pain, urticaria, and dizziness. Serious adverse reactions were hypersensitivity, chills, fatigue, dizziness, and increased body temperature.
In clinical studies of patients being treated with Privigen for chronic ITP, the most common adverse reactions seen in >5% of subjects were headache, elevated body temperature, positive DAT, anemia, nausea, epistaxis, vomiting, increases in conjugated and unconjugated bilirubin, decreased hematocrit, and increased blood lactate dehydrogenase. A serious adverse reaction was aseptic meningitis syndrome (AMS).
Treatment with Privigen might interfere with a patient’s response to live virus vaccines and could lead to misinterpretation of serologic testing. Use in pregnant women only if clearly needed. In patients over 65 or in any patient at risk of developing renal insufficiency, do not exceed recommended dose and infuse Privigen at the minimum rate practicable.
For more information about Privigen, please see full prescribing information.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.