General Updates | Why has the uptake of gene therapy in hemophilia been less than expected?

There have been several impactful business decisions made in the last several months in the field. The manufacturer of the only approved hemophilia A therapy limited their commercial activity to a select few markets, and another manufacturer withdrew their hemophilia B product from the market and decided not to pursue approval for their hemophilia A therapy despite favorable Phase III outcomes. 

Now, this led some to believe that people with hemophilia and their doctors are not interested in gene therapy, which is simply not true. People with hemophilia and their families have really dreamed of a cure since time immemorial, when genes encoding factor VIII and factor IX were cloned. Gene therapy became really synonymous with a cure that was expected to arrive one day. And AAV gene therapy has shown the potential to achieve a functional cure by normalizing clotting factor levels in some, but not all individuals. It is unpredictable who will achieve curative and who will achieve suboptimal therapeutic responses. And it is just one of the many uncertainties that people with hemophilia and their physicians have to consider when making decisions about trying gene therapy or a number of alternative existing therapies, which have evolved along the way and which may offer better understood efficacy and safety profiles. 

It is the responsibility of clinical development programs to demonstrate the additional value of their products. And not many clinical programs have collected or reported data that would enable people with hemophilia and their physicians to differentiate value. So it is, I would say, not surprising that marketing a new treatment product, such as gene therapy using legacy outcomes alone has been insufficient to drive change. I think that a transformative therapy should demonstrate value beyond a zero annualized bleeding rate, especially when this target is possible with other current therapies. Because why should a person with hemophilia take a risk on an uncertain therapy when the manufacturer has not communicated benefit beyond other available therapies? And the hemophilia community has voted with its feet before when new transformative therapies entered the landscape and demonstrated clear improvement over existing therapies. 

Also, gene therapies are very high-priced drugs, let’s face it, which necessitates the establishment of complex reimbursement processes, so that also slows things down. But as far as the enthusiasm and interest in gene therapy, it is definitely still there, and the lesser than expected uptake points to a gap between what the current gene therapies offer and health equity described as a hemophilia-free mind rather than apathy toward curative therapy.

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