Hemgenix, a single-dose cure for a form of the blood-clotting disorder hemophilia, recently set a new world record for the most expensive drug ever at a whopping $3.5 million per dose.
On Tuesday, The US Food and Drug Administration approved Hemgenix, a cutting-edge gene therapy designed to treat adults with hemophilia B, a serious genetic disorder in which people do not produce a critical protein needed to clot blood in case of bleeding. Until now, typical treatments required routine injections to maintain sufficient levels of the missing protein in patients, but thanks to Hemgenix, sufferers of hemophilia B require a single IV infusion to be cured forever. The only problem is that that single dose costs no less than $3.5 million, making Hemgenix the most expensive drug ever.
“Today’s approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia,” Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said.
According to a recent cost-effectiveness analysis by the Institute for Clinical and Economic Review, an independent nonprofit research organization, a fair price for Hemgenix would be between $2.93 million and $2.96 million, which is still ridiculously prohibitive for the average person.
Still, despite its steep price, many believe that Hemgenix has a chance to be successful, for two reasons: existing therapies for hemophilia B are also incredibly expensive, and people with the condition live in constant fear of bleeds that could cost them their lives.
Gene therapies have been getting more expensive over the years. Back in 2018, we wrote about Luxturna, a single-dose gene therapy treatment for inherited blindness, which at the time was the world’s most expensive drug. It cost “only” $850,000.
To claim the title of ‘world’s most expensive drug’, Hemgenix dethroned Zynteglo, a treatment for the blood disorder beta thalassemia, priced at $2.8 million.