Australia’s Therapeutic Goods Administration has approved Sanofi’s efanesoctocog alfa, marketed as Altuviiio, for the treatment and prophylaxis of bleeding episodes in adults and children with haemophilia A, giving Australian clinicians access to a factor VIII replacement therapy with a materially longer duration of action than prior standard-of-care options. The approval adds the therapy to the Australian Register of Therapeutic Goods, clearing the way for listing discussions with the Pharmaceutical Benefits Advisory Committee.

Haemophilia A results from a deficiency or dysfunction of clotting factor VIII, leaving patients prone to spontaneous and trauma-related bleeding into joints and soft tissue. Standard factor VIII replacement products typically require infusions two to three times weekly to maintain protective clotting activity, a treatment burden that has long been cited by patients and haematologists as a barrier to consistent adherence. Efanesoctocog alfa is engineered with a von Willebrand factor-derived fusion component that allows it to circulate independently of endogenous von Willebrand factor, extending its half-life to three to four times that of conventional factor VIII products and enabling once-weekly intravenous dosing while maintaining factor activity levels well above the bleed-risk threshold for most of the dosing interval.

The TGA’s decision follows the therapy’s approval pathway in the U.S. and Europe, where efanesoctocog alfa has been adopted as a preferred option for patients seeking to reduce the frequency of prophylactic infusions without sacrificing bleed protection. Clinical trial data supporting the global filings showed an annualized bleed rate reduction that compared favorably against patients’ own historical rates on prior prophylaxis regimens, with the majority of trial participants experiencing zero treated bleeds during the observation period.

For Australian sponsors and the broader clinical research sector, the approval is a reminder of how the TGA’s regulatory pathway, which frequently follows or runs in parallel with FDA and EMA review rather than requiring fully independent data packages, continues to shorten the lag between global and local access for rare disease therapies. That alignment has increasingly made Australia an attractive site for haematology and rare disease trial activity more broadly, with sponsors citing the country’s centralized ethics review processes and access to treatment-naive patient populations as practical advantages when designing multi-region development programs. Contract research organizations operating in the Australian and New Zealand haemophilia and rare bleeding disorder space have reported growing sponsor interest in leveraging local treatment centers for both regulatory and real-world evidence generation following approvals of this kind.

Sanofi said it expects to begin supply to Australian haemophilia treatment centers in the coming months, with pricing and subsidized access to be determined through the standard PBAC listing process. Approximately 3,000 Australians live with haemophilia A, according to the Australian Haemophilia Centre Directors’ Organisation.