US regulators have approved CSL Behring’s hemophilia B gene therapy, a one-time infusion that frees patients from regular treatments but costs $3.5 million a dose, making it the most expensive drug in the world. world.
CSL Behring’s Hemgenix, given once, reduced the number of bleeding events expected over the course of a year by 54%, according to a pivotal study of the therapy. It also freed 94% of patients from long and expensive infusions of factor IX, which is currently used to control the life-threatening disease.
“Although the price is a bit higher than expected, I think it has a chance of succeeding because 1) existing drugs are also very expensive and 2) hemophilia patients live in constant fear of bleeding,” said Brad Loncar, a biotechnology investor. and Managing Director of Loncar Investments. “A gene therapy product will be attractive to some.”
Gene therapies can dramatically improve a range of devastating conditions by correcting their underlying causes. Novartis AG’s Zolgensma for babies with spinal muscular atrophy was priced at $2.1 million when approved in 2019, while Bluebird Bio Inc.’s Zynteglo for the blood disorder beta-thalassemia, s was $2.8 million earlier this year.
Pricing has been an issue for new drugs, with the high costs of drugs like Biogen Inc.’s Alzheimer’s drug Aduhelm in the US, and Bluebird’s Zynteglo in Europe contributing to their commercial downfall.
Although there have been advances in the treatment of hemophilia, the steps needed to prevent and treat bleeding can erode patients’ quality of life, said Peter Marks, director of the Center for Biologics Evaluation and Research at the United States Food and Drug Administration. Hemgenix represents significant progress in the development of innovative therapies for people affected by the disease, he said.
Traditional treatment for hemophilia infuses missing proteins, called clotting factors, which the body needs to form clots and stop bleeding. Hemgenix works by delivering a gene capable of producing the missing clotting factors to the liver, where it starts working to make the Factor IX protein.
The gene therapy will be manufactured in Lexington, Massachusetts by uniQure NV, which sold the rights to market Hemgenix to CSL Behring in 2020. According to uniQure, approximately 16 million people in the United States and Europe have hemophilia B. Hemophilia A is more common and affects about five times as many people.
(Except for the title, this story has not been edited by NDTV staff and is published from a syndicated feed.)
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