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BioMarin’s hemophilia A gene therapy further delayed amid new FDA questions

June 1, 2022

BioMarin’s commercial aspirations for its hemophilia gene therapy remain on ice after the FDA lobbed additional questions for the company to address, pushing back an expected approval submission to September.

BioMarin was vague on what the FDA is asking for, only specifying that the agency had requested additional details and analyses rather than more studies. The company also pointed out that updated three-year safety data of its phase 3 GENEr8-1 study will become available during the likely review period, although it was unclear whether the FDA would be requesting this.

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