BioMarin’s Momentum Grows With Twin Blockbuster Plans
Dwarfism Drug Data Look Promising
The frontrunner in hemophilia A gene therapy has been hit by data doubts, but this and achondroplasia drug could deliver in 2020.
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During BioMarin’s earnings call Feb. 21, CEO Bienaime indicated the company may be looking to price its valoctocogene roxaparvovec gene therapy at $2m-$3m, as it would represent a savings over the annual cost of infused clotting factor therapy.
The company has come a long way since gene therapy flop Glybera, and claims best-in-class status for viral vector.
Japanese company joins the ranks of big pharma buying up gene therapy pioneers