Hemophilia gene therapy real-world data starts to clarify durability
Approved factor VIII and factor IX gene therapy products are accumulating real-world durability data that defines the addressable-population reality.
Approved hemophilia A (valoctocogene roxaparvovec) and hemophilia B (etranacogene dezaparvovec, fidanacogene elaparvovec) gene therapies have been on market long enough to start generating multi-year durability data. Initial uptake has been measured rather than rapid; payer-channel work, neutralising antibody screening, and patient-and-clinician hesitation about durability all bound the addressable population. The next 12 to 18 months of real-world data clarify the long-term commercial trajectory.
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