Immediately following the FDA’s clearance, the Pennsylvania-based pharmaceutical company CSL Behring released the $3.5 million price tag, arguing that the drug would ultimately result in cheaper healthcare expenditures since patients will have fewer bleeding episodes and need fewer clotting medicines.
According to a research quoted by the National Library of Medicine, Hemgenix is the most costly medication in the world, easily surpassing Novartis’ Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs close to $2 million per dose and is likewise a single-dose drug.
Like the bulk of American pharmaceuticals, the cost of the new treatment will mostly be covered by insurance rather than the patient through both private plans and public programmes.
After decades of study, medications that can alter or fix genetic defects are beginning to transform how cancer and other rare inheritable diseases are treated.
Hemgenix is the first such treatment for haemophilia and several other pharmaceutical companies are working on gene therapies for the condition’s more common type, 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.
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