CSL Behring's Afstyla has been approved in the US as a long-lasting treatment option for patients with the bleeding disorder haemophilia A.
Afstyla is a novel recombinant factor VIII single-chain therapy, the first and only single-chain product for haemophilia A specifically designed for long-lasting protection from bleeds with two to three times weekly dosing.
Specifically, it can now be used in both adults and paediatric haemophilia A patients for routine prophylaxis to reduce the frequency of bleeds, on-demand treatment and control of bleeds, and the perioperative management of bleeding.
In clinical trials, patients undergoing prophylaxis with Afstyla experienced a median annualised spontaneous bleeding rate of zero, while a "strong safety profile" was also demonstrated with no inhibitors observed, the firm noted.
The "FDA's approval of the first recombinant single-chain therapy that offers long-lasting hemostatic efficacy provides an important new treatment option for patients and healthcare providers as it has been specifically designed for increased molecular stability and duration of action," said study investigator Lisa Boggio, Assistant Professor of Internal Medicine, Hematology and Oncology, clinical director of the Rush Hemophilia and Thrombophilia Center.
"Afstyla offers patients an opportunity for excellent efficacy with a strong safety profile and twice-weekly dosing - potentially helping patients to fit treatment into their active lives," she noted.
Regulators in Europe, Switzerland and Australia are also currently reviewing license applications for the therapy.