FDA blocks much-anticipated BioMarin hemophilia gene therapy

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FDA rejects potentially game-changing hemophilia A gene therapy BioMarin over concerns it might not really be a one-and-done lifetime treatment

The U.S. Food and Drug Administration's rejection late Tuesday means the San Rafael, California-based company will have to complete an ongoing late-stage patient study, likely delaying possible approval till late in 2022.

The company added that FDA concluded differences between the results of a small, early-stage study and interim data from the late-stage study left unclear how long the therapy's effect would last. The gene therapy works by using an inactivated virus, created in a lab, to deliver to liver cells a working gene via a one-time IV infusion meant to enable the body to produce FVIII on its own.


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'...Over Financial Concerns Regarding This One-and-Done Treatment.'

This is why we must pass the Promising Pathway Act

Its time to stop playing god. We dont know the effects of this stuff. How about starting at curing the common cold first...

Gene therapy are we better than nature ?

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