TGA approval for CSL Behring's Haemophilia A Treatment

Thursday, 20 April, 2017

CSL Behrin has announced that the Australian Therapeutic Goods Administration (TGA) has approved AFSTYLA [lonoctocog alfa], a recombinant single chain coagulation factor VIII (rFVIIISingleChain) in patients with haemophilia A (congenital factor VIII deficiency).

The treatment is indicated in all patients with haemophilia A for: routine prophylaxis to prevent or reduce the frequency of bleeding episodes; control and prevention of bleeding episodes; perioperative management (surgical prophylaxis). AFSTYLA is “the first and only single-chain recombinant factor VIII product to treat haemophilia A”. The single chain design provides sustained protection from bleeds with a strong binding affinity to Von Willebrand factor. In clinical trials, patients undergoing prophylaxis with AFSTYLA experienced a median annualised spontaneous bleeding rate (AsBR) of zero with two to three times weekly dosing. It was demonstrated to be well tolerated with no patients discontinuing the study due to adverse events.

For an estimated 81% of severe haemophilia A patients who receive prophylaxis every second day or three times a week (McRae S, 2016), the switch to extended dosing intervals with AFSTYLA will reduce the number of injections required for prophylaxis therapy without compromising control of bleeding.

“The Australian Haemophilia Centre Director’s Organisation (AHCDO) views the development of new effective clotting factor concentrates as a further step forward in the management of our patients with haemophilia,” said Dr Simon McRae, Consultant Haematologist and Chairman of AHCDO. “The ability to prevent the vast majority of bleeding events with less frequent infusions has the potential to improve long-term outcomes in individuals with haemophilia.”

Approval of AFSTYLA is based on results from the AFFINITY clinical development program. This includes Phase I through to Phase III open-label, multicentre studies evaluating the safety and efficacy of the treatment in children and adults (ages 1 to 61 years) with severe haemophilia A.

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