Data Bytes: Comparing Factor VIII levels of hemophilia A gene therapies

Hemophilia A is expected to be the next market where gene therapies will create competition, but long-term durability remains a key unknown.

While the BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) therapy valoctocogene roxaparvovec, or “valrox,” maintains Factor VIII levels in severe hemophilia patients above the thresholds for moderate and severe disease, those levels, measured in IU/dL by chromogenic substrate assay, have gradually decreased over four years...