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  • Writer's pictureJohn Q Leonard

Welcome to Age of One-Shot Miracle Cures That Can Cost Millions

World’s Most Expensive Drug Approved to Treat Hemophilia at $3.5 Million a Dose


Recently CSL Behring was granted FDA Approval of Hemophilia B Gene Therapy that will be the most expensive drug on the market for any disease. Proponents of Hemgenix claim that the drug is a powerful one-shot "cure" for a life-threatening and debilitating disease.


Hemgenix is one-time gene therapy administered by IV infusion that cuts the number of bleeding events and freed 94% of patients from regular treatments, making it the most expensive medicine in the world.


“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) hemophilia 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 was priced at $2.1 million when it was approved in 2019, while Bluebird Bio Inc.’s Zynteglo for the blood disorder beta thalassemia came in at $2.8 million earlier this year.


Pricing has been an issue for novel medicines, with high costs for drugs like Biogen Inc.’s Alzheimer’s drug Aduhelm in the US and Bluebird’s Zynteglo in Europe contributing to them becoming commercial busts. An assessment by the Institute for Clinical and Economic Review, a nonprofit that evaluates medical costs, found that a fair price for Hemgenix would be in the range of $2.93 million to $2.96 million.


Welcome to Age of One-Shot Miracle Cures That Can Cost Millions



This blog post was summarized from a report by Michelle Fay Cortez on November 22, 2022

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