Historic hemophilia nod paves way for world's most costly medicine

23 November 2022
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In a landmark regulatory first, Dutch gene therapy company uniQure (Nasdaq: QURE) has secured US approval for its one-time hemophilia B treatment, Hemgenix (etranacogene dezaparvovec-drlb).

Further development and commercialization will be taken forward by licensor CSL Behring, part of Australia's CSL Limited (ASX: CSL), after the firm  secured global rights at a potential cost of over $2 billion.

The US Food and Drug Administration granted approval for the treatment of certain adults with hemophilia B, including those who currently use factor IX prophylaxis therapy.

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