Shares of BioMarin Pharmaceutical(NASDAQ:BMRN) are down 35% at 1:41 p.m. EDT because the U.S. Food and Drug Administration issued a complete response letter for the biotech's hemophilia A gene therapy, valoctocogene roxaparvovec (valrox).

A complete response letter is the FDA's euphemism for a rejection letter. The review is complete, but the agency needs more information before it can approve the treatment.

In BioMarin's case, it seems the FDA moved the goalpost for approval.

The agency had previously told BioMarin that it could file the marketing application with interim data from the phase 3 clinical trial that showed the treatment increased the activity of Factor VIII, the clotting factor that's missing in patients with hemophilia A. Valrox puts the Factor VIII gene into patients' cells, which allows their blood to clot after they're cut.

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