US: FDA approves most expensive drug ever, costs $3.5 million-per-dose

The Pennsylvania-based pharmaceutical company CSL Behring published the $3.5 million price tag immediately after the FDA granted its approval, claiming that the medicine would ultimately lower healthcare costs because patients would experience fewer bleeding events and require fewer clotting therapies. 

The price of Hemgenix, which easily surpasses Novartis’ Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs close to $2 million per dose and is also a single-dose medication, makes it the most expensive medication in the world, according to a study cited by the National Library of Medicine. 

The cost of the new treatment, like that of the majority of American medications, will primarily be covered by insurers, not by the patient, both through private plans and public programmes.

After decades of research, gene treatments have started to change how cancer and other uncommon inheritable diseases are treated by using drugs that can alter or correct genetic abnormalities. 

The first such treatment for haemophilia is Hemgenix, and a number of other pharmaceutical companies are developing gene treatments for the disorder’s more prevalent variant, haemophilia A. 

The liver, which produces the clotting protein, receives a functional gene from Hemgenix. 

According to the FDA, haemophilia B affects approximately 1 in 40,000 individuals and accounts for 15 per cent of those who have the condition.

(with inputs from agencies)