Global biotherapeutics leader CSL Behring announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation for Afstyla [Recombinant Human Coagulation Factor VIII, Single Chain] for patients with haemophilia A. Afstyla, CSL Behring’s novel, recombinant factor VIII therapy for adults and children, is the first and only single-chain product for haemophilia A. It is specifically designed for protection from bleeds with two or three times weekly dosing and low unit consumption at both dosing regimens. In clinical trials, Afstyla demonstrated a strong safety profile with no inhibitors observed in previously treated patients undergoing prophylaxis.

“For 100 years, CSL has focused on researching and developing innovative therapies that meet the treatment challenges patients face,” said Dr. Andrew Cuthbertson, chief scientific officer and R&D director, CSL Limited. “CHMP’s positive opinion for Afstyla moves us one step closer to bringing this novel treatment option to haemophilia A patients in the European Union. Once approved, Afstyla will provide adults and children with a therapy that delivers on our promise to develop and bring to market innovative specialty biotherapies that help patients live full lives.”

Primarily affecting males, haemophilia A is a congenital bleeding disorder characterised by deficient or defective factor VIII. People with haemophilia A may experience prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs. According to the World Federation of Hemophilia, about 1 in 10,000 people are born with haemophilia, the majority of whom have haemophilia A.

Afstyla (also known as rVIII-Single Chain) for haemophilia A is CSL Behring’s recombinant single-chain factor VIII specifically designed for greater molecular stability and longer 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 factor VIII activity with the option of twice weekly dosing.

Afstyla is approved in the US. In addition to the European Union, regulatory agencies in markets around the world, including Switzerland and Australia, are currently reviewing CSL Behring’s marketing applications for Afstyla. Regulatory submissions for Afstyla are based on results from the AFFINITY clinical development program. Data from AFFINITY were recently published in the American Society of Hematology’s publication Blood and data comparing the pharmacokinetics of Afstyla and octocog alfa in patients with severe haemophilia A were recently published in The World Federation of Hemophilia’s journal Haemophilia.