Valrox could become BioMarin’s largest product, says Jefferies

BioMarin is set to report topline three-year Phase 2 data for hemophilia A gene therapy valrox by June 7, including clotting factor VIII levels, annualized bleeding rate, and factor VIII usage, Jefferies analyst Eun Yang tells investors in a research note. The analyst estimates potential valrox market size at $12B in the U.S. and $10B in Europe. As such, valrox, if efficacy is durable, has the potential to be the largest product for BioMarin, says the analyst. After surveying 26 U.S. hematologists, Yang believes doctors are focused more on the clinically relevant bleeding rate for valrox utilization, in line with BioMarin’s position, versus the Street’s focus on factor VIII levels. If valrox is approved and 10%-30% of patients receive valrox thru 2023, the analyst sees 11%-30% upside to her current price target of $125 for BioMarin shares. She keeps a Buy rating on the name.

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