BioMarin Says It's Got The Hemophilia Therapy Data For Approval And Value

The company will begin discussions with regulators in the US and Europe for a gene therapy for hemophilia A based on the results of new data, with a launch targeted for late 2020. Durability remains a question.

Red blood cells. Blood elements - red blood cells responsible for oxygen carrying over, regulation pH blood, a food and protection of cages of an organism. - Illustration

BioMarin Pharmaceutical Inc. will begin regulatory discussions in the US and Europe for its gene therapy for severe hemophilia A based on interim data from an ongoing Phase III trial and updated three-year data from an ongoing Phase I/II trial.

The latest data for BioMarin’s gene therapy for severe hemophilia A raise some questions about its long-term durability, but BioMarin...

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