The US Food and Drug Administration (FDA) has approved a new treatment for a rare blood clotting disease, 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 what was reported by the website.Science Alert“.
The drug, called Hemgenix, is a gene therapy for hemophilia B, a rare genetic disease that causes low blood clotting. More serious symptoms include spontaneous and recurrent bleeding episodes that are difficult to stop.
Hemophilia B affects more men than women, and while an exact number is difficult to come by, it is estimated that nearly 8,000 men in the United States currently suffer from the disease for life.
But the costs of lifelong treatment are exorbitant. For those with severe symptoms, a routine and expensive treatment regimen is needed, and its effectiveness begins to wane over time.
Researchers estimate the life cost per patient with moderate to severe hemophilia B at $21 to $23 million, according to the report.
Two studies have so far tested the drug’s efficacy and safety. And in one study of 54 participants with severe or moderate hemophilia B, researchers found good results for the drug, which reduced the need for routine alternative therapies currently available to patients.
Side effects included headaches, flu-like symptoms and enzyme elevations in the liver, all of which must be carefully monitored going forward with treatment.
“Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advance in developing 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 is not yet clear if this gene therapy is a cure for hemophilia B, but initial results are promising.
The European Medicines Agency and drug regulators in the UK and Australia are also now reviewing gene therapy for use.
