New most expensive drug in the world: what is it for and why does Hemgenix cost so much

The drug Hemgenix recently received approval from the regulatory agency for food and drugs in the United States (FDA, its acronym in English) to be marketed in the country.

It is a gene therapy to treat haemophilia B, the least common and most dangerous form of the disease that makes it difficult for blood to clot.

CSL Behring, the drug’s maker, has priced a single-dose treatment at $3.5 million in the United States – making it by far the most expensive drug in the world.

what is it for

Approximately one in every 40,000 people has hemophilia B, a disease that affects men more, as many women are asymptomatic.

Hemophilia, which is divided into types A and B, is caused by an error in the genetic code that causes the individual not to produce essential proteins for blood clotting, which can lead to fatal bleeding.

People with hemophilia B are unable to produce a protein in their blood plasma called factor IX.

Until now, these patients needed to receive factor IX injections, usually several per week.

The new therapy consists of a laboratory-developed virus that carries a gene to produce clotting factor IX.

Two studies with 57 adult patients with hemophilia B (from moderate to severe) demonstrated the effectiveness of Hemgenix, which provides for a single application in combating the disease.

“Subjects treated with Hemgenix showed an increase in mean factor IX levels and hemostatic protection lasting for two years,” said CSL Behring.

While it will take decades to prove whether the cure is permanent, for now these results are considered solid.

The FDA said, for its part, that the new treatment “enables people living with hemophilia B to produce their own factor IX, which may reduce the risk of bleeding.”

record price

The price of US$ 3.5 million for a single dose is “higher than any medicine on the market”, David Rind, medical director of the Institute of Clinical Review and (ICER), an independent entity that assesses the value of drugs and other medical products.

Hemgenix is ​​more expensive than other highly valued single-dose gene therapy drugs such as Zynteglo ($2.8 million) and Zolgensma ($2.1 million), which treat beta thalassemia major and muscle wasting. infantile spinal cord, respectively.

“The price we consider fair for Hemgenix is ​​between US$ 2.93 million and US$ 2.96 million”, says the director of ICER.

“We see that manufacturers charge high prices for gene therapies, especially if they appear to be effective.”

But what justifies the astronomical price of the drug?

why is it so expensive

In 2020, CSL Behring paid $450 million to the initial developer, uniQure, to license the therapy and market the drug, according to Forbes magazine.

The biotechnology company said in a statement that the price “was determined taking into account the clinical, social, economic and innovative value that this new gene therapy represents.”

And he argued that, as it is a single-dose therapy, its price is lower than that of the chronic injection treatments used so far – which, according to estimates, could cost up to US$ 20 million per person over a lifetime.

This “could offer significant cost savings to the overall healthcare system,” the company added.

CSL Behring has reserved the exclusive right to distribute this drug in the US market for the next seven years, the high cost of which will be largely covered by the government and health insurers.

In the US, the Executive encourages pharmaceutical companies to stimulate the production of therapies for rare diseases, and does not impose price caps on medicines.

This means that the US pays between two and six times more for prescription drugs than other countries in the world, according to the specialized publication Science Alert.

The price of Hemgenix when it is available in health systems in other countries has not yet been disclosed – but, according to experts, it may be lower.

This text was originally published here.