16 January 2020 - Valrox would be first gene therapy approved in U.S. for inherited type of haemophilia.
BioMarin Pharmaceutical is exploring pricing its experimental gene therapy for haemophilia patients between $2 million and $3 million if it is approved, which could make it the most expensive drug in the world.
The company hasn’t yet announced a price for the therapy, but Chief Executive Jean-Jacques Bienaimé said in an interview at the J.P. Morgan Healthcare Conference that the drugmaker has spoken to insurers, who have indicated they are comfortable with the range.