approved the most expensive drug in the world. It’s a gene therapy – time.news

from Silvia Turin

One dose will cost $3.5 million, but giving just one will save the NHS millions of dollars while giving hope to people with haemophilia B

The Federal Drug Administration (FDA) of the United States has just approved the most expensive drug in the world: 3.5 million dollars per dose.

The rare disease

The medicine, called Hemgenix , a treatment of gene therapy for haemophilia B, which is a rare genetic disorder that causes impaired blood clotting. also called Christmas disease, after the patient in whom the condition was first observed. characterized by the lack, in the plasma, of an indispensable factor for
coagulation
called factor IX or PTC (Plasma Thromboplastin Component) or, indeed, Christmas factor.
Transmitted with a recessive gene located on the X chromosome, it affects only individuals of the same gender male with a frequency of 1 in 40,000 (15% of haemophilia sufferers)while women, who act as carriers by transmitting the disease to their sons, are not subject to it.

How does it work

Hemgenix, an intravenous gene therapy for adults that it is injected once in a single dose. The drug is delivered in the body via a viral-based vector designed to deliver DNA to liver cells. The genetic information contained in the drug reaches the cells and supplies the instructions for building the coagulation protein which is missing, Factor IX. So far, two studies have tested the efficacy and safety of Hemgenix. In a study of 54 participants with severe or moderately severe haemophilia B, the rate at which patients developed uncontrolled bleeding decreased by more than 50% compared to baseline. Side effects included headaches, flu-like symptoms and liver enzyme elevations.

High cost that saves money

Compared to the exorbitant cost, nearly 8,000 men in the United States currently suffer from the disease. In those with severe symptoms, a treatment regimen is needed by periodic and expensive dripwhich over time can begin to decrease in effectiveness: the researchers, writes Sciencealert, estimate that the cost of adult life for each patient with moderate to severe hemophilia B is about 21-23 million dollars.
Hemgenix, on the other hand, is only given once. classified as an orphan drug because it treats only a limited number of patients. It could still save millions in medical bills, improving the lives of these patients. It is not yet clear whether this treatment will be a cure for haemophilia B, but early results are promising.

Hemophilia B

The main feature of the disease is the great facility to
severe bleeding
and sometimes unstoppable even after trauma or minor injuries such as bruises, intramuscular injections, dental avulsions. From classical hemophilia (or hemophilia A) the type B differs in the lesser severity of symptoms and course, and in the different nature of the defect (haemophilia A due to factor VIII or antihaemophilic globulin deficiency). The disease has a chronic course, generally the haemorrhagic manifestations diminish, however, after the age of twenty-five.

The previous record holder of most expensive drug in the world was another one-off form of gene therapy for the treatment ofspinal muscular atrophy (SMA). The European Medicines Agency (EMA) and drug regulatory agencies in the UK and Australia are also now reviewing treatment for haemophilia B.

November 26, 2022 (change November 26, 2022 | 11:48 am)

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