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At $3.5 Million A Dose, This Is The World’s Most Expensive Medicine


At $3.5 million a dose, this is the most expensive drug in the world

Gene therapies can improve a wide range of devastating conditions by correcting their underlying causes

U.S. regulators approved CSL Behring’s hemophilia B gene therapy, a one-time infusion that spares patients from conventional treatment but costs $3.5 million a dose, making It became the most expensive drug in the world.

A pivotal study of this therapy found that CSL Behring’s Hemgenix, administered only once, cut the number of expected bleeding episodes by 54% over the course of a year. It also freed 94% of patients from the time-consuming and costly infusion of Factor IX, which is currently being used to manage the potentially fatal condition.

Brad Loncar, a biotech investor, said: “Although the price was a bit higher than expected, I think it has a good chance of succeeding because 1) existing drugs are also very expensive and 2 ) patients with hemophilia always live in fear of bleeding. and the chief executive officer of Loncar Investments. “A gene therapy product will appeal to some people.”

Gene therapies can dramatically improve a wide range of devastating conditions by correcting their underlying causes. Novartis AG’s Zolgensma for infants with spinal muscular atrophy cost $2.1 million when approved in 2019, while Bluebird Bio Inc.’s Zynteglo. for the blood disorder beta thalassemia cost $2.8 million earlier this year.

Price is an issue for new drugs, with high costs for drugs like Biogen Inc.’s Alzheimer’s drug Aduhelm. in the US and Bluebird’s Zynteglo in Europe contributed to their commercial bankruptcy.

While there have been advances in the treatment of hemophilia, measures are needed to prevent hemophilia, said Peter Marks, director of the US Food and Drug Administration’s Center for Biological Evaluation and Research. Preventing and treating bleeding can reduce a patient’s quality of life. Hemgenix represents important progress in developing innovative therapies for people affected by the disease, he said.

Traditional hemophilia treatments infuse missing proteins, called clotting factors, that the body needs to form blood clots and stop bleeding. Hemgenix works by introducing a gene that can make missing clotting factors into 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 commercialization rights to Hemgenix to CSL Behring in 2020. About 16 million people in the United States and Europe have hemophilia B, according to a report. uniQure. Hemophilia A is more common, affecting five times as many people.

(Except for the title, this story has not been edited by NDTV staff and is published from an aggregated feed.)

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