FDA has approved Afstyla, the first and only recombinant Factor VIII single chain therapy for hemophilia A
The FDA ( U.S. Food and Drug Administration ) has approved Afstyla [ Antihemophilic Factor ( Recombinant ), Single Chain ], a 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 have 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.
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.
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% of bleeds in adult and adolescent patients and 96% of bleeding events in pediatric patients were effectively controlled with no more than two infusions of Afstyla weekly; 81% of bleeds in adult and adolescent patients and 86% of bleeding events in pediatric patients were effectively controlled by only one infusion.
The majority of bleeding events treated with Afstyla ( 94% in adults and adolescents; 96% 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. ( Xagena )
Source: CSL Behring, 2016