The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for Afstyla (lonoctocog alfa) for the treatment and prevention of bleeding in adults and children with hemophilia A.
Developed by CSL Behring, Afstyla is a single-chain recombinant human factor VIII product that will be marketed as a powder and solvent for solution to be delivered as an injection or infusion.
The U.S. Food and Drug Administration (FDA) approved Afstyla in May this year for the treatment of adult and pediatric patients with hemophilia A.
The FDA approval was based on data from the AFFINITY clinical development program, published in the journal Blood, under the title “Efficacy and safety of rVIII-SingleChain: results of a phase I/III multicenter clinical trial in severe hemophilia A.”
Data from these clinical trials show that the drug prevented and controlled bleeding on demand and during surgical procedures in adults and children with hemophilia A.
The proposed full indication is: “Treatment and prophylaxis of bleeding in patients with hemophilia A (congenital factor VIII deficiency). Afstyla can be used for all age groups.”
“Afstyla has demonstrated a higher affinity for von Willebrand factor than full-length recombinant factor VIII. Von Willebrand factor stabilizes factor VIII and protects it from degradation,” an EMA representative said in a press release.
Afstyla’s most common adverse effects include hypersensitivity, dizziness, paresthesia (abnormal tingling sensation), rash, and pyrexia (fever).
Afstyla will be available by prescription. CSL Behring suggests that only physicians experienced in the treatment of hemophilia prescribe the new treatment.
Soon, CSL will publish a summary of product characteristics containing detailed recommendations for the use of Afstyla. After the European Commission grants final marketing authorization to the product, the summary will be available in all official European Union languages in the European public assessment report.
Single-chain design provides a more resistant protection against bleeding. This design also allows increased binding affinity to other blood components (von Willebrand factor, or VWF) which keeps factor VIII in circulation longer and protects it from degradation.
Afstyla replaces missing factor VIII, a coagulation factor required for effective hemostasis. Afstyla has demonstrated a higher affinity for VWF than full-length recombinant factor VIII.