The TGA has approved CSL Behring's IDELVION (albutrepenonacog alfa) for use in patients with haemophilia B (congenital factor IX deficiency).
The therapy is indicated in all patients with haemophilia B for: routine prophylaxis to prevent or reduce the frequency of bleeding episodes; control and prevention of bleeding episodes; and, control and prevention of bleeding in the perioperative setting.
IDELVION is a long-acting albumin fusion protein linking recombinant coagulation factor IX with recombinant albumin and is appropriate for use in all age groups.
The company said it is the first and only Australian registered factor IX therapy that delivers high-level protection with up to 14-day dosing for appropriate patients.
"It helps patients maintain factor IX activity levels over a long period of time, facilitating greater patient freedom from frequent infusions. For patients receiving twice weekly factor IX, a switch to extended dosing intervals with IDELVION reduces the monthly factor IX consumption needed for prophylaxis therapy without comprising control of bleeding," it said.
“IDELVION provides excellent bleeding control by maintaining factor IX activity levels above 5 percent over a prolonged period of time,” said Loretta Croker, general manager of CSL Behring Australia and New Zealand.
“IDELVION delivers on CSL’s 100 year promise to develop and provide innovative specialty biotherapies that patients need and want. We are committed to bringing IDELVION to Australian patients with haemophilia B and particularly excited about the positive impact this long-acting therapy can have on their lives,” she said.
“The Australian Haemophilia Centre Director’s Organisation (AHCDO) views the development of effective long-acting clotting factor concentrates as a major step forward in the management of our patients with haemophilia,” said Dr Simon McRae, consultant haematologist and chairman of AHCDO. “The ability to maintain clotting factor levels above a level that prevent the vast majority of bleeding events with less frequent infusions has the potential to improve long-term outcomes in individuals with haemophilia.”
According to the company, approval of IDELVION is based on results from the PROLONG-9FP clinical development program. PROLONG-9FP includes Phase I through Phase III open-label, multicenter studies evaluating the safety and efficacy of IDELVION in children and adults (ages 1 to 61 years) with haemophilia B (factor IX levels ≤ 2 per cent).
“I have seen first-hand the benefits IDELVION has had on children with haemophilia B, said Dr Julie Curtin, senior staff specialist in haematology, The Children’s Hospital at Westmead and an investigator in the PROLONG-9FP clinical development program. “IDELVION has enabled children on regular treatment with factor IX to reduce the frequency of infusions without increasing their risk of bleeding. For a child to only need an injection every 1-2 weeks is a good step forward in the management of haemophilia B which I welcome and I am sure my patients will welcome this improvement too.”
Gavin Finkelstein, President of the Haemophilia Foundation Australia said, “The community is very pleased to see the approval of new treatment opportunities for people with haemophilia B.”
Haemophilia treatments are funded through the National Blood Authority.