The haemophilia B gene therapy developed by CSL Behring was given the green light by US regulators. This one-time infusion frees patients from ongoing treatment. It is the most expensive drug in the world at $3.5 million (PKR 78 crore) per dosage.
CSL Behring’s Hemgenix, administered just once, cut the number of bleeding events expected over the course of a year by 54%, a key study of the therapy found. It also freed 94% of patients from time-consuming and costly infusions of factor IX, currently used to control the potentially deadly condition.
“While the price is a little higher than expected, I do think it has a chance of being successful because 1) existing drugs are also very expensive and 2) haemophilia patients constantly live in fear of bleeds,” said Brad Loncar, a biotechnology investor and chief executive officer of Loncar Investments.
“A gene therapy product will be appealing to some.”
Gene therapies can dramatically improve a range of devastating conditions by fixing their underlying causes. Novartis AG’s Zolgensma for babies with spinal muscular atrophy priced at $2.1 million when it approved in 2019. Whereas, Bluebird Bio Inc.’s Zynteglo for the blood disorder beta thalassemia came in at $2.8 million earlier this year.
