CSL/UniQure’s Hemgenix To Enter Uncertain EU Market After Regulatory Nod
The firms’ one-time hemophilia B gene therapy has been approved in the EU just months after a US nod, but concerns remain about its prospects on a market that has seen several setbacks for other gene therapies.
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CSL Behring said it remains confident that a market access agreement for its hemophilia B gene therapy Hemgenix can be reached after England’s HTA body NICE rejected the product because of uncertainties over its long-term benefit and cost-effectiveness.
An awaited update on early data for a CAR-NK therapeutic shows disappointing efficacy and duration, but a different chemotherapy conditioning regimen for lymphodepletion is showing early promise in acute myeloid leukemia.
Public Company Edition: Seven companies revealed $2.1bn worth of royalty financings in the first quarter of 2023 and the deals keep coming in Q2, with uniQure monetizing Hemgenix royalties for up to $400m. Also, Legend grossed $350m in a follow-on offering and Athenex is liquidating via bankruptcy.