And drug capable of being able to cure one rare genetic disease but paying Millions of dollars.
Hemgenix is a latest generation drug, approved on November 22 by the Fda (Food and drug administration), the US regulatory agency, produced by the pharmaceutical company Csl Behring. The drug is the result of long years of research into the development of a therapy that can cure thehaemophilia ba rare genetic blood disorder that involves prolonged bleeding episodes in the event of trauma or wounds and, in the most serious cases, even spontaneous bleeding and haemorrhage, without a clear cause.
A gene therapy that will cost patients over three million dollars for a single dosegaining the title of most expensive drug in the world, not without criticism for the exorbitant amount. However, according to the results, the drug would have the advantage of eliminating the bleeding episodes caused and associated with the disease in over half of the patients tested.
And while the drug is still under considerationEma (European Medicines Agency) it is advisable to understand how this drug works and above all how much it costs and why so much. Here’s everything you need to know.
Hemgenix, how does the drug work to treat haemophilia B?
The most expensive drug in the world, Hemgenixwill cure thehaemophilia ba rare genetic disease which involves a coagulation disorder caused by a mutation in the gene for the factor IXprotein needed for stop bleeding in case of injuries.
According to the data reported by the ISS, this disease strikes a person his 30,000 and hits the mensince it is factor IX, while women can be healthy carriers, even if it is estimated that around 10-25% still show mild symptoms and in rare cases even severe symptoms.
If usually the treatment ofhaemophilia b involves the administration of a coagulating factor, with Hemgenix everything would change. In fact, as Wired also explains, the drug, administered by intravenous injection, is an adenoviral vector that carries the missing gene for coagulation. Once arrived at the liver this produces the needed protein to prevent bleeding episodes.
And according to the two clinical studies, i 57 patients (men) affected by severe haemophilia Bor moderately severe, have seen shrink by 54% the rate of bleeding events annually, as well as registering an increase in the activity of the coagulating factor. While the contraindications include headaches, flu-like symptoms and increased liver enzymes. An excellent result for researchers who see the way open to new treatments and new research in this field.
Hemgenix, the drug to treat haemophilia B: here’s how much it costs
What if Hemgenix is celebrated in the field of research, it is true that it is true that patients can not afford to pay a therapy dal cost so high. In fact, as also explained by Bloomberg, Hemgenix it would cost $3.5 million per dosewinning the title of drug most expensive in the world.
Really a lot if you think that patients could undergo multiple doses over the years. According to a study by theInstitute for clinical and economic reviewan organization that looks after the costs of drugsand right price” for Hemgenix it should be between $2.93 million and $2.96 million per dose: still a lot for the patients.
Yet second Brad Loncarbiotech investor and chief executive officer of Loncar Investments, although the price is quite high could have a ‘chance of success’ because existing drugs to treathaemophilia b however, they are very expensive and patients living with the fear of possible bleeding could see Hemgenix as an opportunity for a new life. All that remains is to wait for the response of the EMA, which has not yet pronounced itself on the new drug.