The appropriate pricing of potentially curative gene therapies remains a contested question, but BioMarin Pharmaceutical Inc. seems ready to push a high boundary. Execs indicated during the firm’s earnings call Feb. 21 that it is looking to price the Phase III hemophilia A candidate valoctocogene roxaparvovec (BMN 270 or val-rox) at $2m-$3m, which would still represent savings compared to frequent infusions of Factor VIII therapies.
The first gene therapy approved in the US, Spark Therapeutics Inc.’s Luxturna (voretigene neparvovec-rzyl) treatment for a rare inherited form of blindness, was expected to come with a $1m price tag, but the company launched with a price of $850,000. (Also see "The First US Gene Therapy Maker Innovates On Pricing And Reimbursement" - Scrip, 3 January, 2018