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