The most expensive drug in the world has been approved in the US – the Federal Medicines Agency has approved a new drug for a rare blood clotting diseasewhich costs $3.5 million.
The drug is called “hemgenix” and is a genetic therapy for hemophilia B, a rare disease that causes impaired blood clotting. The most serious symptoms include spontaneous and repeated episodes of bleeding, which are difficult to stop.
Haemophilia B is usually more common in men than in women. Estimates show that almost 8,000 men in the USA currently suffer from this disease – the “Science Alert” portal reports.
The main drug currently used to treat hemophilia B in the US gives patients a much-needed clotting factor, but its lifetime treatment costs are high. Persons with severe symptoms require a routine and expensive treatment regimen, the effectiveness of which may weaken over time, reports Blic, writing by Srna.
Scientists estimate that the lifetime cost of each patient with moderate to severe hemophilia B is about $21 million to $23 million. Treatment costs in Britain are cheaper than in the US, or elsewhere in Europe, but still run into tens of millions of dollars per patient over their lifetime.
“Hemgenix”, on the other hand, is a disposable intravenous product that is given in one dose at a lower price.
The product is delivered into the body via a virus-based vector, which is engineered to deliver the DNA to target cells in the liver. That genetic information is then replicated by cells, spreading the “instructions” for the production of a clotting protein known as factor nine.
Two studies have so far examined the effectiveness and safety of this drug. In one study among 54 actors with severe or moderate hemophilia B, scientists found increased levels of factor nine, which reduced the need for otherwise available replacement therapies.
After receiving the gene therapy, the rate at which patients developed uncontrolled bleeding dropped by more than 50 percent compared to the baseline rate.
Side effects of taking Hemgenix include headaches, flu-like symptoms, and increased liver enzymes.