€1.5m Price Tag Could Spell EU Success For BioMarin’s Hemophilia Gene Therapy
First Hemophilia A Gene Therapy Approval
The approval is a breakthrough for BioMarin, which is hoping to seize market share from Factor VIII replacement therapies and Roche’s Hemlibra.
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Sanofi can now expect an FDA decision in early 2023 on efanesoctocog, setting it up for a battle with Roche’s Hemlibra and BioMarin’s gene therapy Roctavian, which was just approved in the EU.
Bluebird bio will not see revenue for Zynteglo until it is infused into beta-thalassemia patients, which will take months due to the ex vivo gene therapy’s complex manufacturing and quality control process.
Phase III success for efanesoctocog alfa sets up a filing in the near future, and a chance to seize market share from existing Factor VIII products, as well as Roche’s Hemlibra.