Experimental hemophilia drug could be the most expensive in the world

Anicka Slachta | | Cardiovascular Business | Vascular & Endovascular
Researchers found millions of heart failure patients who qualify for standard, guideline directed medical therapies, but did not receive it due to poor health literacy, limited access to care and medication costs.

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.

BioMarin Explores Pricing Experimental Gene Therapy at $2 Million to $3 Million

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

The company filed for regulatory approval with the FDA in December. The earliest they expect to make a decision on price is probably late August, according to Bienaimé.

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Anicka Slachta

After graduating from Indiana University-Bloomington with a bachelor’s in journalism, Anicka joined TriMed’s Chicago team in 2017 covering cardiology. Close to her heart is long-form journalism, Pilot G-2 pens, dark chocolate and her dog Harper Lee.

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