Experimental Hemophilia Drug Could Be The Most Expensive In The World

Biotech company BioMarin Pharmaceutical is considering a price point of between $2 million and $3 million for its novel experimental gene therapy for hemophilia patients, the Wall Street Journal reported Jan. 16.

If approved at that price, valoctocogene roxaparvovec, also known as valrox, would beat out Novartis’ Zolgensma as the most expensive drug in the world. Zolgensma, approved in the U.S. as a therapy for patients with spinal muscular atrophy, costs a hefty $2.1 million per round of treatment.

Valrox would also be the first gene therapy approved for an inherited type of hemophilia that’s characterized by a deficiency of an essential clotting protein. Chief Executive Jean-Jacques Bienaimé told the Journal payers “seem to be comfortable” around the $2 million to $3 million mark, noting the overall cost of care for hemophilia patients will likely drop. Bienaimé said BioMarin is open to alternative payment structures, including pay-for-performance, but insurers have suggested they’d pay up front for valrox.

“At the end of the day, where I stand today, I think most of them will pay up front,” he said.