Hemgenix (hemophilia drug)

2022: Product Announcement

In late November 2022, the United States approved gene therapy for hemophilia from CSL Behring, a drug called Hemgenix that exempts patients from regular treatment. The drug costs $3.5 million per dose, making it the most expensive drug in the world.

CSL Behring's Hemgenix, which is given just once, reduces the amount of bleeding expected during the year by 54%, a key study of this therapy found. Hemgenix also exempted 94% of patients from time-consuming and time-consuming factor IX infusions, which as of November 2022 is used to combat the deadly disease.

Treatment of hemophilia for $3.5 million. The most expensive drug in the world came out

Pricing has become a problem for new drugs, and high prices for drugs such as the Alzheimer's drug Aduhelm developed by Biogen. in the US and Zynteglo developed by Bluebird in Europe, led to them becoming commercially unsuccessful.

{{quote 'Although the price is slightly higher than expected, I believe that the drug has a chance of success because, firstly, existing drugs are also very expensive, and secondly, hemophiliacs constantly live in fear of bleeding! The gene therapy product will be attractive to some, "said Loncar Investments biotechnology investor and chief executive officer Brad Loncar. }} Despite advances in hemophilia, measures needed to prevent and treat bleeding can reduce patients' quality of life, FDA Center for Biologics Evaluation and Research Director Peter Marks said. CSL Behring represents an important advance in the development of innovative therapies for people affected by the disease.

Gene therapy will be produced in Lexington, by uniQure NV, which sold the commercialization rights to Hemgenix from CSL Behring in 2020. According to uniQure, about 16 million people in the United States and Europe have hemophilia B, and hemophilia A is more common, affecting about five times as many people.^[1]

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