Gene Therapy Shows Promise for Hemophilia, But Could Be Most Expensive U.S. Drug Ever

July 20, 2020

(NPR) – One of the biggest questions is the possible cost. BioMarin Pharmaceutical Inc. of San Rafael, Calif., the company that developed the gene therapy, says the treatment could cost as much as $3 million per patient, which would make it the most expensive drug ever approved. “It’s just outrageous,” says Peter Bach, who studies drug prices at Memorial Sloan Kettering Cancer Center in New York.