The US Federal Drug Administration (FDA) has approved a new treatment for a rare blood clotting disorder. Each dose will cost 3.5 million dollars (about 3.3 million euros), making it the most expensive drug in the world.
the medicine, named Hemgenix, is a treatment for hemophilia B, a rare genetic disorder that causes reduced blood clotting. More severe symptoms include spontaneous and repeated bleeding that is difficult to stop.
As reported by the hair Science Alert, hemophilia B tends to be more common in men. Estimates suggest that around 8,000 men in the United States (US) suffer from the disease.
The main medication used to treat hemophilia B in the US gives patients the necessary clotting factor, but they are high. In severe cases, an expensive treatment regimen is required, which over time it starts to lose effectiveness🇧🇷
The researchers estimate that the cost of treating each patient with moderate to severe hemophilia B is about $21 to $23 million ($20 to $22 million) over a lifetime. In the UK they are cheaper than in the US or elsewhere in Europe, but still high.
Hemgenix is a one-time intravenous product. It is transported into the body via a viral-based vector, created to deliver DNA to target cells in the liver. This genetic information is replicated by cells, spreading out the instructions for a clotting protein, known as Factor IX.
So far, the effectiveness and safety of Hemgenix has been tested in two studies. In one investigation, among 54 participants with severe or moderately severe haemophilia B, levels of Factor IX activity were found to increase, reducing the need for routine replacement therapies.
After receiving gene therapy, the rate at which patients developed uncontrolled bleeding decreased by more than 50%🇧🇷 Side effects included headaches, flu-like symptoms and increased liver enzymes.
“Despite advances in the treatment of hemophilia, preventing and treating bleeding episodes can have a negative impact on an individual’s quality of life,” said Peter Marks, director of the FDA’s Center for Biological Evaluation and Research, quoted in a announcement of the entity.
“Today’s approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies for those suffering from a high burden of disease associated with this form of hemophilia,” he continued.
As the article underlined, it is not yet known whether this treatment is a cure for hemophilia B, but initial results are promising.
For serious but rare conditions like hemophilia B, the FDA has a special designation to encourage medical investigation. Hemgenix, for example, is classified as an “orphan drug” because it will only treat a small number of patients.
As part of this designation, the manufacturer of Hemgenix, CSL Behring, has exclusive rights to the US market for the next seven years.
US government incentives to encourage scientific research is a useful way to drive innovation in rare diseases that would otherwise fall by the wayside, but this policy is a “double-edged sword”.
It also means that the US market bears the cost of supporting drug monopolies, while other countries, which impose restrictions on drug prices, reap the benefits of research. Currently, the US pays between two and six times more for drugs than other nations.
The previous record holder for the most expensive drug was another form of single-dose gene therapy to treat spinal muscular atrophy🇧🇷 Each dose costs 2 million dollars (around 1.9 million euros).