The US Food and Drug Administration (FDA) has approved a new treatment for a rare blood clotting disorder, which is an expensive treatment and is the most expensive in the world.
The cost of the drug is 3.5 million US dollars, but despite this, its price is reasonable compared to its effectiveness in treating a rare disease, according to the website.Scientific alarm“.
The drug, called Hemgenix, is a gene therapy for hemophilia B, a rare genetic disorder that causes low blood clotting. More serious symptoms include spontaneous and recurring bleeding episodes that are difficult to stop.
Hemophilia B affects more men than women, and while it’s hard to come up with an exact number, it’s estimated that nearly 8,000 men in the United States currently have the disease for life.
But the costs of permanent treatment are exorbitant. For those with severe symptoms, a routine and expensive treatment regimen is required, and its effectiveness begins to wane over time.
Researchers estimate the cost of living per patient with moderate to severe hemophilia B at $21 to $23 million, according to the report.
Two studies have so far tested the efficacy and safety of the drug. And in a study of 54 participants with severe or moderate haemophilia B, researchers found good results for the drug, which reduced patients’ need for currently available routine alternative therapies.
Side effects included headaches, flu-like symptoms and liver enzyme elevations, all of which need to be closely monitored as treatment progresses.
“Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for those with a high disease burden associated with this type of hemophilia,” said Peter Marks, MD, director of the FDA’s Center for Biological Evaluation and Research.
It’s not yet clear whether this gene therapy is a cure for haemophilia B, but early results are promising.
The European Medicines Agency and drug regulators in the UK and Australia are also reviewing the use of gene therapy.
