“It’s been absolutely brilliant and life-changing for me,” says [Jack] Grehan, 26, of Billinge in North West England. He received an experimental gene therapy in 2017 that, at least for now, has eliminated his need for regular [blood clotting] injections. “I can just go about my day and not have to worry.”
Based on experiences like Grehan’s, the company that developed the therapy is seeking approval in Europe and the United States to start selling the first gene therapy for hemophilia. That’s generating excitement among patients, patient advocates and doctors.
Others are more cautious.
“This is really exciting, but also raises a lot of questions,” says Meg Bradbury, director of research at the Hemophilia Federation of America, a patient advocacy group.
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.
Company officials defend the possible price, however. It currently costs hundreds of thousands of dollars a year to treat each hemophilia patient. The gene therapy would, researchers hope, be a one-time treatment that lasts a lifetime.