CSL and uniQure unveil three-year data for one-time hemophilia B gene therapy that costs $3.5M

With its $3.5 mil­lion price tag, CSL Behring and uniQure’s one-off he­mo­phil­ia B gene ther­a­py Hem­genix is one of the world’s most ex­pen­sive ther­a­pies. On Fri­day, three-year fol­low-up from its reg­is­tra­tional study of­fers a new look at the drug’s cu­ra­tive po­ten­tial.

The HOPE-B study test­ed a dose of Hem­genix in 54 men with the rare bleed­ing dis­or­der. The treat­ment se­cured an FDA ap­proval in No­vem­ber 2022 on the ba­sis of topline re­sults.

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