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CSL Behring Presents Pivotal Efficacy Data for AFSTYLA® In Adolescents and Children with Hemophilia A at the World Federation of Hemophilia 2016 World Congress | ||
By: PR Newswire Association LLC. - 27 Jul 2016 | Back to overview list |
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ORLANDO, Fla., July 27, 2016 /PRNewswire/ -- CSL Behring today presented data from a Phase III pivotal study of AFSTYLA® [Antihemophilic Factor (Recombinant), Single Chain] in hemophilia A patients less than 12 years of age at the XXXII International Congress of the World Federation of Hemophilia (WFH) in Orlando, Fla. The results showed that AFSTYLA, used prophylactically across different treatment intervals (including two to three times weekly), achieved low annualized spontaneous bleeding rates (median AsBR of 0.00) and low annualized bleeding rates (median ABR of 3.69) in previously treated children and adolescents with hemophilia A. Overall, the efficacy of AFSTYLA to treat bleeding events in patients on prophylaxis or on-demand regimens was rated as excellent or good by investigators in 96.3 percent of all bleeding events (total of 347 treated events). "The results show that AFSTYLA provided robust protection to both prevent and manage bleeds in children and adolescents with hemophilia A," said Professor Ingrid Pabinger-Fasching, M.D., of the Medical University of Vienna, Austria, and lead investigator of the study. "AFSTYLA fills an important need for the pediatric hemophilia A community, helping to meet their treatment challenges and offering an opportunity for excellent efficacy with a strong safety profile and more convenient dosing." The study was part of the Phase I/III AFFINITY clinical program, which included two completed pivotal studies and one ongoing open-label, multicenter extension study evaluating the safety and efficacy of AFSTYLA in children, adolescents, and adults with hemophilia A. The current study results reported at WFH included data on more than 5,200 exposure days in previously treated pediatric patients with severe hemophilia A (factor VIII levels <1%). Patients had more than 50 previous exposure days to factor VIII products prior to enrollment. During the study, patients received either prophylactic (n=81) or on-demand (n=3) treatment with AFSTYLA according to WFH dosing recommendations. The median time on study was 5.6 months. Among patients in the prophylaxis group, 54 percent were dosed twice weekly, and 30 percent were dosed three times weekly. The most common adverse reactions were nasopharyngitis (common cold), arthralgia (joint pain), cough, and headache. There were no serious adverse events, and no patients developed inhibitors while receiving AFSTYLA. 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. Regulatory agencies in Europe, Switzerland, and Australia are currently reviewing CSL Behring's license applications for AFSTYLA. Additional AFSTYLA Data Presented at WFH At WFH, investigators also presented the effects of an intensified prophylaxis regimen with AFSTYLA (50 IU/kg three times weekly) that was prescribed by the investigator to eradicate a pre-existing low titer inhibitor in a patient enrolled in the pediatric study (Poster #89). The individual had approximately 27 exposure days to factor VIII therapy in the year prior to enrollment and about 100 exposure days overall. He was enrolled into the study with a pre-existing low-titer inhibitor that was not reported at screening. The results showed that the patient's inhibitor titer continuously decreased, dropping below the cut-off for positivity (<0.6 BU/mL) after three months and remaining negative through the end of the study. In addition, all bleeding episodes (total of seven treated) were well-controlled with AFSTYLA. The patient continues to be treated with prophylactic AFSTYLA in the extension study. "For 100 years CSL has developed and delivered innovative treatments for patients with serious medical conditions and, today, we are pleased to present at the WFH 2016 World Congress new AFSTYLA data from our AFFINITY clinical development program," said Dr. Andrew Cuthbertson, Chief Scientific Officer and Director of R&D, CSL Limited. "The collective results build upon previous data demonstrating the efficacy of this innovative therapy for routine prophylaxis, on-demand treatment and perioperative bleeding management in hemophilia A. As the first and only FDA-approved single-chain recombinant factor VIII product, AFSTYLA delivers improved protection from bleeding with less frequent dosing and has an excellent safety profile." About Hemophilia A About AFSTYLA® For more information about AFSTYLA, including full prescribing information in the U.S., please visit http://labeling.cslbehring.com/PI/US/Afstyla/EN/Afstyla-Prescribing-Information.pdf. U.S. Only: AFSTYLA Important Safety Information
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 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. In the U.S., 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 (>0.5%) trials were dizziness and hypersensitivity. About CSL Behring 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. Contact:
SOURCE CSL Behring |
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