FDA chief Gottlieb is building a regulatory speedway to accelerate gene therapy development
In a rallying cry for gene therapy, FDA Commissioner Scott Gottlieb says he’s determined to clear the pathway for drug developers in a move to accelerate the first wave of gene therapies pointed to the market.
The first therapeutic area to benefit from new surrogate endpoints will be hemophilia, Gottlieb said — immediately ringing a bell for companies like Spark Therapeutics $ONCE, Pfizer $PFE, BioMarin $BMRN and uniQure $QURE, which are developing cures for both versions of the bleeding disorder. Under the yet-to-be-announced guidelines, factor production may in some cases be sufficient as a measure of benefit.
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