What’s the lesson of BioMarin’s FDA rejection?
BioMarin Pharmaceutical shocked the biotech world yesterday with its disclosure that the FDA had rejected its gene therapy for hemophilia A, a treatment widely expected to win approval and become a revolutionary product.
The news, as BioMarin tells it, is that the FDA moved the goalposts on its gene therapy after previously agreeing to a path toward approval. The agency now wants to see two additional years of data on the treatment’s durability, something BioMarin said it learned only by reading the rejection letter. More on that here.
In the meantime, the rest of the gene therapy field is left to consider what this regulatory decision means for other products. Competitors in the space told STAT that BioMarin’s FDA situation appears to be company specific, but outside experts cautioned that it could herald a shift in how the agency considers one-time treatments with irreversible effects. Here’s more on that.
The news, as BioMarin tells it, is that the FDA moved the goalposts on its gene therapy after previously agreeing to a path toward approval. The agency now wants to see two additional years of data on the treatment’s durability, something BioMarin said it learned only by reading the rejection letter. More on that here.
In the meantime, the rest of the gene therapy field is left to consider what this regulatory decision means for other products. Competitors in the space told STAT that BioMarin’s FDA situation appears to be company specific, but outside experts cautioned that it could herald a shift in how the agency considers one-time treatments with irreversible effects. Here’s more on that.
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