Gene therapy company uniQure’s (NASDAQ:QURE) partner CSL has received approval for Hemgenix for the treatment of adults with haemophilia B in the U.S.
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CSL had licensed global rights for the gene therapy from QURE in 2021 and is now solely responsible for its development and commercialization.
Importantly, Hemgenix has now become the first gene therapy across the globe to be approved for this indication which is a rare and lifelong bleeding disorder.
QURE is responsible for the global manufacturing of the therapy and has received ~$500 million from CSL so far. It further stands to gain up to $1.5 billion in milestone payments from CSL.
Further, Hemgenix is also being evaluated by other healthcare regulators.
Next, the company has priced this historic therapy at $3.5 million. This makes the miracle one-shot cure, the most expensive medicine worldwide, according to Bloomberg.
QURE shares have now rallied 72% in the past six months and the Street still sees a further 1.5x upside in the stock.
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