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Record breaking $3.5 million single-dose for hemophilia B

The first hemophilia B gene therapy has been approved by the US Food and Drug Administration. The treatment price per dose for this drug is $3.5 million, making it the most expensive drug in history.


The gene therapy HEMGENIX® was developed by uniQure to help treat individuals with hemophilia B. It is a rare genetic disease most commonly found in men and impacts the bloods ability to clot,  due to a lack of or ineffective factor IX. The single dose therapy allow patients to produce IX removes the reliance and need for prophylactic therapy.

HEMGENIX® has now been taken over by CSL Behring, a world-leading biotechnology company that provides innovative medicines. They are working towards making the treatment available to individuals with hemophilia B within the U.S.

Bill Mezzanotte, Head of Research & Development and Chief Medical Officer of CSL had this to say: “With HEMGENIX, we now offer a comprehensive portfolio of innovative medicines for hemophilia B, giving people living with the condition more choice in treatments and better and more durable control over their disease.”

An evaluation of HEMGENIX is still being taken by other regulatory agencies along with other clinical trials. We look forward to hearing more about the progress of this new gene therapy.

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