Pharmaceutical Technology

CSL’s haemophilia B therapy Hemgenix scores win authorisation in Canada

The world’s most expensive drug, Hemgenix, has been granted authorisation in Canada after being approved in other regions.

Health Canada has authorised CSL Behring’s Hemgenix, further expanding the reach of the haemophilia B gene therapy after approvals in the US, Europe and the UK.

The FDA approval was granted in November 2022 based on the findings from the Phase III HOPE-B trial. The one-off infusion comes with a list price of $3.5m a dose, making it the most expensive drug in the world.

GlobalData forecasts predict that the global market for haemophilia A and haemophilia B treatments will grow to $14.7bn by 2030, up from $9.4bn in 2020. Gene therapies such as Hemgenix are expected to be significant drivers for this growth.

GlobalData is the parent company of Pharmaceutical Technology.

Haemophilia B is a rare, lifelong bleeding disorder caused by a mutation in the F9 gene, resulting in insufficient production of factor IX, a crucial protein needed for proper blood clotting. Hemgenix works by enabling the body to continuously produce factor IX, employing adeno-associated virus (AAV) technology to introduce the Padua gene variant of Factor IX (FIX-Padua) into liver cells promoting the production of factor IX proteins that are significantly more active than normal.

Traditional treatments for haemophilia B prophylaxis therapy, require strict, lifelong infusion schedules and may not entirely prevent spontaneous bleeding episodes or the associated pain, joint damage, and swelling.

The Phase III HOPE-B trial (NCT03569891), which drove the authorisation, evaluated the safety and efficacy of AMT-061 in patients with severe to moderately severe haemophilia B. The study enrolled 54 subjects, making it the largest gene therapy trial for haemophilia B to date.

Positive data from the study showed that 94% of patients discontinued prophylactic treatments and remained free of continuous routine prophylaxis therapy after 24 months.

CSL research and development head Bill Mezzanotte said: "We believe Hemgenix has the potential to change the treatment paradigm for people living with haemophilia B and the healthcare professionals who treat them, as it addresses the cause of the condition – faulty factor IX gene expression."

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