25 million with one injection?! The record for 'the world's most expensive drug' is still controversial...

Yesterday, the US Food and Drug Administration (FDA) approved a new drug, Hemgenix, for marketing, causing a global media sensation.This is an injection agent, the dose is not large, one shot down can cure hemophilia B, but the price is ridiculously high:A dose of 3.5 million US dollars (about 25.02 million yuan)...

Such a sky-high price made Hemgenix instantly set a new record and became the most expensive drug in the world.Why is a small injection so expensive?This has to start with the condition it treats - hemophilia B.

Hemophilia B is a hemorrhagic genetic disease, because of a congenital genetic defect, the liver is unable to produce clotting factor IX, blood clotting disorders occur, and a slight trauma will cause bleeding.

The medical community has deduced that the "cursed hemorrhagic disease" that afflicted many male descendants of Queen Victoria in the late 19th and early 20th centuries is a serious type B hemophilia.

About 15% of hemophilia patients have hemophilia B, and the average person has a 1 in 40,000 chance of developing it, the vast majority of whom are men, and women with this type of genetic defect usually have no symptoms.Hemophilia B is both a rare disease and a chronic disease once thought to be incurable.

In the past, the common treatment for patients with hemophilia B was regular injections of clotting factors to maintain a certain concentration of clotting factors in the blood and prevent bleeding.Such treatment not only requires a long-term burden of expensive drugs, but also patients are always on the lookout and live in anxiety about injury and bleeding.

Therefore, in the view of scientists, in order to cure hemophilia B, it is necessary to find a way to repair the defect at the genetic level, in order to hope to completely cure hemophilia B.

CSL Behring, a biopharmaceutical company in Pennsylvania, began investing a lot of manpower and material resources ten years ago to develop gene therapy for hemophilia B.

In the past ten years, hundreds of researchers have repeatedly studied and carried out many clinical verifications in many patients, and the world's first and only gene therapy injection Hemgenix for the treatment of hemophilia B was born.

Hemgenix's clinical data is pleasantly surprised:With only one dose unit injected at a time, the liver cells of adult hemophilia B patients can produce coagulation factor IX, and the bleeding symptoms are greatly reduced or even disappeared, and 94% of patients can completely get rid of regular injection of coagulation factors!
It can be said that Hemgenix is the first genetic drug that can cure hemophilia B once and for all.

After officially obtaining approval from the Food and Drug Administration on Tuesday, the company's CEO happily declared:This is a milestone in the history of hemophilia B treatment, allowing patients who have been waiting for more than 20 years to look forward to the dawn of a radical cure.

Scientists and pharmaceutical companies are celebrating the approval of the new drug Hemgenix, but the media and patients have expressed great concern about Hemgenix's record high price.After all, a shot costs $3.5 million, which is not affordable for the average person...

Some experts insist that the pricing is reasonable:Pharmaceutical companies have invested a lot of money in the development of drugs, hemophilia B is a rare disease, Hemgenix is not used by a particularly wide range of people, to recover the cost, can only set such a high price.Experts believe that even so, $3.5 million a shot is still cost-effective for patients, because the existing type B hemophilia drugs on the market are themselves expensive, need to be used for a long time, and the cost accumulated over the years is also huge.And Hemgenix can solve the problem in one go, saving the patient the torment of the second half of his life.

According to other experts, the reality is:In recent years, rare disease drugs such as Hemgenix have repeatedly hit sky-high prices and have become a trend.

Before Hemgenix, the world's most expensive drug was Zolgensma, a drug launched by Novartis in 2019, a drug for infants and young children under the age of 2 with spinal muscular dystrophy (SMA).Similar to Hemgenix, Zolgensma is also a genetic drug, which also has the same effect once and for all, and the price is equally ridiculously expensive, costing $2.125 million for a shot...

In May this year, some media made a ranking of the world's top ten most expensive drugs, including Zolgensma, and almost all of the top drugs are for the treatment of rare diseases.For example, Zokinvy, which treats cognitive progeria, sells for as much as $1.07 million.

The successful development of rare disease drugs such as Hemgenix has brought good news to patients on the one hand, and has also caused great controversy on the other hand:Some people believe that such expensive drugs, the vast majority of patients simply cannot afford it, often need the government and society to bear, is a test for the medical insurance system of various countries.

In this regard, the Japanese government provides a reference, last year, the Japanese government announced the inclusion of Zolgensma in medical insurance, which can be regarded as solving the urgent needs of some patients' families.

In addition, some experts believe that pharmaceutical companies invest a lot of manpower and material resources to develop rare disease drugs, and most of the time they can get compensation and high returns from the government.Once pharmaceutical companies determine that rare disease drugs are profitable, they will put more resources into the research of rare disease drugs, inevitably ignoring the research of epidemic diseases such as diabetes and hypertension, and the research and cure of these common diseases will be further delayed...

I hope that in the future, people can find a way to promote the smooth development of drugs without rushing out of outrageous drugs.