4hw.com.cn: "I'm not the God of medicine" has been well received since its release. The film takes Lu Yong, the first person to buy anti-cancer drugs, as the prototype, to tell the story of CML patients who, under the pressure of high drug prices, embarked on the road of purchasing foreign generic drugs overseas. So do you know what other expensive drugs besides CML are around us? Take a look at the seven most expensive drugs in the world.
Seventh soliris
Annual cost: 3.6 million yuan
Not long ago, soliris (eculizumab) was the most expensive drug in the world, now ranked seventh, with an annual cost of nearly 3.6 million yuan.
Soliris treats two rare diseases: paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). PNH destroys the red blood cells of patients, with an estimated 8000-10000 patients in North America and Europe. AHUS affects the body's immune system in a way that can lead to serious complications, including blood clots. Only 1500 patients have been diagnosed with the disease, but up to 41000 aHUS patients worldwide may be diagnosed.
Sixth place actimmune
Annual cost: 3.79 million yuan
Actimmune (Interferongamma-1b, interferon γ -1b) can treat two rare diseases: chronic granulomatosis (CGD) and severe malignant osteoporosis (SMO). Horizon's pricing for actimmune is 3.79 million yuan per patient per year.
The drug is a kind of interferon gamma; protein produced by a biological method. The incidence of CGD in the United States was 1 /20, while the incidence rate of SMO was 1 /25 million.
Fifth place carbaglu
Annual cost: 3.88 million yuan
Carboglu (carboglutamic acid) is used to treat patients with urea circulatory disorders (UCDs). However, unlike ravivicti, carbaglu is specifically targeted at N-acetylglutamate synthetase (nags) deficiency. The drug's developer, recordati rare diseases, priced carbaglu at no less than 3.88 million yuan per patient per year.
Carbaglu is the only drug approved by FDA for the treatment of hyperammonemia in patients with nags deficiency of all ages. There are seven types of UCD. Although there are few patients of each type, the cases caused by nags deficiency are the most rare. As the diagnosis of nags defects is insufficient, the actual number of people affected by UCD is still unknown.
Fourth place lumizyme
Annual cost: 4.15 million yuan
Lumizyme (recombinant glucosidase alpha;) is used to replace a missing or deficient enzyme in patients with Pompeii disease. Pompeii disease is a genetic disease caused by the accumulation of a sugar called glycogen in body cells. The drug, marketed by Sanofi, costs more than 4.15 million yuan a year.
The incidence of Pompeii's disease is estimated at 1 out of every 40 thousand live births, with a incidence rate of about 1/1.4 million among African Americans. In 2006, Sanofi's Myozyme was approved for treatment of Pompeii disease. However, when the company is ready to expand the capacity of the bioreactor that produces Myozyme, FDA requires a separate approval process. This led to the approval and listing of lumizyme in 2010.
Third place spinraza
Annual cost: 4.97 million yuan
Spinraza is a new member of the most expensive prescription drug for spinal muscular atrophy (SMA). It is an antisense oligonucleotide lead compound that binds to mutated RNA.
The drug was originally developed by Ionis pharmaceuticals and was approved by the FDA on December 23, 2016.
SMA is a rare and often fatal genetic disease that affects muscle strength and movement. Spinraza is the first drug approved for the treatment of the disease. The cost of treatment in the first year is 4.97 million yuan, but it can be reduced to 2.5 million yuan a year later, because more injections are needed in the first year than in subsequent years.
Second place ravicti
Annual cost: 5.27 million yuan
Ravivicti (glycerophenylbutyrate) is a kind of medicine for the treatment of congenital urea circulatory disorder (UCD). It works by removing ammonia and preventing the harmful accumulation of ammonia in the body. The accumulation of this toxic substance can lead to brain damage and death. According to horizon's pricing, the annual cost per patient is nearly 5.27 million yuan.
In the United States, an estimated 2000 people suffer from urea cycle disorders. Ravivicti can treat 7 types of UCD, but whether ravivicti can treat N-acetylglutamate synthetase (nags) deficiency safely and effectively is a rare type, which has not been determined.
First place, glybera
Annual cost: 6.63 million yuan
Glybera (alipogeneiparvovec) is a gene therapy drug, which can be used to treat adult patients diagnosed with familial lipoprotein lipase deficiency (lpld). It is a rare genetic disease, resulting in a large amount of fat accumulation in the blood, which can cause severe pancreatitis. The drug, developed by Dutch pharmaceutical company uniqure, costs 663 per year.
Glybera won approval from the European Medicines Agency in 2012. Currently, the drug is not directly available to Americans, and the FDA requires additional clinical trials, which Uniqure has decided not to conduct further.
The seven most expensive drugs are just the tip of the iceberg. According to 2015 data, more than 90 kinds of prescription drugs cost more per year than nearly 500000 yuan of "Lenin" in the film. We have a long way to go to reduce the price and sales of drug research and development and benefit more people.