- What is now the most expensive drug in the world is the first and only one-time gene therapy treatment for adults with hemophilia B.
- During clinical trials the drug was able to reduce the rate of annual bleeding.
- It has just been approved by the FDA and has a cost of 3.5 million dollars.
Over the last half century, an exponential increase in life expectancy has been achieved globally. People are living longer and longer, and one of the reasons is the development of new drugs against a wide range of diseases. In fact, there is a drug that has just been approved and broke the record with which it became the most expensive in the world.
First of all, it is necessary to mention the one that held this feat until before 2022. It is about Zolgensma, which was designed for the treatment of spinal muscular atrophy. It was developed by the Novartis laboratory and is administered intravenously.
Its commercialization began in 2019 and the main indication is that only children under two years of age can receive it. While the complete treatment of this drug is two million dollars.
For a couple of years he was considered the most expensive drug in the world but now it will no longer be so. It is all due to the US Food and Drug Administration (FDA) approval of Hemgenix which now holds the new record.
What does it work for?
Developed by CSL Behring, it is indicated against hemophilia B and has a price of 3.5 million dollars. Never before has a formula had such a high cost to the public.
Today, we approved a gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. https://t.co/LR6v9KXxM8 pic.twitter.com/lN8iue1mXK
—US FDA (@US_FDA) November 22, 2022
With respect to its indications, so far it is the First and only one-time gene therapy treatment for adults with hemophilia B. It is recommended for people who are currently using factor IX prophylaxis therapy or have current or history of life-threatening bleeding. Also for those who have repeated severe spontaneous bleeding episodes.
For his part, during clinical trials the drug was able to reduce the rate of annual bleeding. While 94% of patients discontinued factor IX prophylaxis and remained prophylaxis-free after treatment.
as long as Hemgenix is a one-time gene therapy product that is administered as a single dose by IV infusion. It consists of a viral vector that carries a gene for coagulation factor IX. The gene is expressed in the liver to produce the Factor IX protein and thus limit bleeding episodes.
Adverse reactions it causes
Regarding the most common adverse reactions, the following are mentioned: elevations in liver enzymes, headache, mild infusion-related reactions, and flu-like symptoms.
For now, the one who is now the The world’s most expensive drug is now available in the United States. Now it is expected that the manufacturer will begin the procedures to request its sanitary approval in other countries and may be available to more people.
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