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BioMarin resubmits its hemophilia gene therapy to the FDA

September 30, 2022

Roctavian’s road back to the FDA has been long, as BioMarin has had to gather additional data in support of its therapy. Delays and new safety concerns further hampered the company’s resubmission plans.

The treatment is for people with severe hemophilia A, a genetic disease that leaves people with little or no clotting protein needed to prevent excessive bleeding.

While there are many treatments that work by replacing this protein artificially, Roctavian would be the first in the U.S. to address the disease’s root cause by replacing the defective gene with a functional copy.


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