BioMarin Sets High Price Tag for Hemophilia Gene Therapy Candidate

If its experimental gene therapy for patients with hemophilia A is approved by the FDA, BioMarin Pharmaceutical is considering pricing valoctocogene roxaparvovec (valrox) between $2 million and $3 million, making it the most expensive treatment in the world.

The world’s current most expensive drug is another gene therapy: Novartis’s Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy for the treatment of spinal muscular atrophy, priced at $2.1 million.

In clinical trials of valrox, patients who received the gene therapy saw their bleeding episodes essentially disappear, dropping from an average of 16.5 events per year to an average of zero. This improvement was sustained for 3 years.

BioMarin CEO Jean-Jacques Bienaimé said insurers have indicated they are comfortable with the proposed price range, comparing it with the lifetime cost of treatment for hemophilia using existing therapies, which he estimates to be about $25 million. However, some patients’ levels of factor VIII fell during the study period, raising concerns about whether the treatment would last a lifetime.

BioMarin filed for FDA approval in December 2019, but the agency hasn’t told the company when it expects to announce its decision. Pfizer and Roche’s Spark Therapeutics are also developing gene therapies for hemophilia A.

Source: The Wall Street Journal, January 16, 2020.