Sanofi nabs TGA approval for next-gen haemophilia A therapy

The Therapeutic Goods Administration (TGA) has approved Sanofi’s first-in-class haemophilia A therapy, Altuviiio (efanesoctocog alfa), marking a notable addition to the treatment landscape for this rare disease.

Haemophilia A is a congenital, lifelong bleeding disorder caused by deficiency or dysfunction of clotting factor VIII, leaving patients susceptible to spontaneous or recurrent bleeding events. In Australia, more than 3,000 people are estimated to live with haemophilia, with haemophilia A accounting for approximately 80% of cases.

As a high-sustained factor VIII replacement therapy, Altuviiio is engineered to deliver extended protection against bleeding episodes in both adults and children living with haemophilia A, with the convenience of once-weekly intravenous dosing. Its extended half-life is three to four times longer than rival therapies.

Dr Stephanie P’Ng, Haematologist at Fiona Stanley Hospital, Chair of the Australian Haemophilia Centre Directors’ Organisation (AHCDO), and an investigator in the Altuviiio XTEND-1 trial said, “Altuviiio represents an important advancement in haemophilia A management. A therapy that can deliver sustained protection with less frequent dosing has the potential to ease treatment burden for patients and support greater confidence in everyday activities.”

Altuviiio enters a competitive haemophilia A treatment landscape already shaped by Roche’s subcutaneous bispecific monoclonal antibody Hemlibra (emicizumab), which was funded under the National Blood Agreement in November 2020. More recently, Pfizer’s Medical Services Advisory Committee (MSAC) application for funding Hympavzi (marstacimab) in this patient cohort was rejected.

The Haemophilia Foundation Australia described the approval as a significant milestone for the haemophilia A community.

“Clinical research has demonstrated the ability to address an important unmet need by offering the convenience of once-weekly dosing with the potential to reduce treatment burden while maintaining effective protection,” Alan Dursun, President of Haemophilia Foundation Australia told Health Industry Hub.

Key efficacy findings from the XTEND-1 trial show that patients receiving weekly prophylactic Altuviiio over 52 weeks achieved a median annualised bleed rate (ABR) of 0, with a mean ABR of 0.71. Exit interviews with trial participants suggest meaningful improvements in physical functioning and joint pain, with 96.6% of patients reporting high satisfaction and 100% preferring Altuviiio over their previous treatments.

While not a head-to-head clinical trial, a matching-adjusted indirect comparison (MAIC) between Hemlibra’s HAVEN 3 trial and XTEND-1 trial indicated improved efficacy with Altuviiio prophylaxis, including significantly reduced rates of any bleeds, any treated bleeds, and treated joint bleeds. Although patient populations across both trials were broadly similar, differences were noted, including a higher baseline burden of joint disease in the HAVEN 3 cohort.

Liz Selby, Country Lead and Head of Pharma at Sanofi ANZ, said “The registration of Altuviiio marks an exciting milestone for the haemophilia community in Australia. Our focus is not only on reducing bleeding episodes, but on helping people living with haemophilia experience greater freedom and confidence in their daily lives.”

She added, “While access pathways here can be complex and lengthy, we are committed to working with urgency alongside government, clinicians and the community to help ensure timely access for those who may benefit. Australian patients should not have to wait longer than in other countries for access to medicines they need.”

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