CSL’s world-first gene therapy heads for MSAC evaluation

Biotech News: CSL’s world-first gene therapy for haemophilia B is scheduled for consideration at the upcoming Medical Services Advisory Committee (MSAC) meeting in August. The Department of Health has issued a directive to CSL Behring to submit a co-dependent application for the anti-AAV5 NAb test to be evaluated in parallel to Hemgenix (etranacogene dezaparvovec).

Haemophilia B is a rare congenital bleeding disorder, affecting approximately 15% of the prevalent population. It stems from deficiencies in coagulation factors due to mutations in clotting factor genes. Currently, patients undergo fortnightly prophylactic infusions of factor IX (FIX) to manage the condition.

Hemgenix, with its staggering price tag of $5 million ($US3.5 million) per dose is the most expensive treatment in the world. Having acquired global licensing rights from UniQure in 2020, CSL Behring has been developing Hemgenix for close to a decade.

Under current standards of care, patients face fortnightly infusion of FIX, a burden that Hemgenix aims to alleviate with its one-time dosing. Available FIX replacement therapies funded via the National Blood Agreement and managed by the National Blood Authority (NBA) include plasma-derived FIX replacement products (e.g. MonoFIX), recombinant products (e.g. BeneFIX) and EHL formulations (e.g. Alprolix).

By significantly reducing or eliminating the need for prophylactic FIX infusions, CSL Behring anticipates a substantial decrease in healthcare resource utilisation, including fewer physician visits, outpatient appointments, emergency room visits, and hospitalisations due to bleeds.

Independent analyses, such as the Institute for Clinical and Economic Review’s assessment, underscore the potential cost-saving benefits of gene therapies like Hemgenix. With projected savings of over $7 million per patient at a price point of $US2.5 million per dose, the economic implications are significant.

Consultation feedback from key stakeholders, including the Royal Brisbane and Women’s Hospital Haemophilia Treatment Centre (RBWH-HTC), Australian Haemophilia Nurses Group (AHNG), Thrombosis and Haemostasis Society of Australia and New Zealand (THANZ) and Haemophilia Foundation Australia (HFA), has largely supported public funding for Hemgenix.

However, concerns raised by these stakeholders pertained to psychological impacts of both response and non-response to treatment, long-term data availability, and post-intervention lifestyle adjustments highlight the complexities inherent in adopting gene therapies.

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