Expensive injections. The most expensive drugs
Expensive medicines, although you are expensive, you are very dear to us. Those whom fate has baffled with very rare diseases could say so about some medicines, produced pharmaceutical industry for patients “forced to be rich.”
From an economic perspective, global drug sales will exceed $1.3 trillion in 2018. This is more than the GDP of 15 countries combined. Eat traditionally inexpensive drugs, accessible to the widest masses, from time to time and in different parts of the planet suffering from the same ailments. But for some people with problems, especially those whose diseases are very rare. So-called orphan drugs designed to treat rare diseases can cost consumers six figures. In many cases, this price becomes essentially the cost of human life.
In countries where insurance medicine dominates, Insurance companies bills for super-expensive drugs are not always paid, forcing patients and their loved ones to incur significant expenses. In some places, men and women have to “work for the pharmacy” for many years. Sometimes they get help non-profit organizations or just random fellow citizens who responded to a request for help. But this is only sometimes.
In 2015, Glybera replaced Soliric as the most expensive drug in the world. The cost of a one-year course of this masterpiece of pharmacology was 1.2 million US dollars. Glybera is needed for people with a deficiency of the enzyme lipoprotein lipase, which regulates lipids in the blood. In Europe, only 1,200 people live with this health disorder, and no more than a million worldwide.
Glybera is available in the form of a solution for intramuscular injections. This product works on the principle of gene therapy, which is an innovation. Glybera was developed in Amsterdam and became the first genetic medicine approved for use in the European Union, where only two per million people suffer from lipoprotein lipase deficiency (LPLD). The disease is accompanied by severe attacks of pancreatitis. It is believed that one injection of Glybera is enough to relieve a patient of LPLD. But the price of such an injection is equivalent to the cost of a miracle; it is not for nothing that the drug was not approved for quite a long time. Only specially trained doctors working in special treatment centers. It is assumed that European residents are ready to pay the pharmaceutical industry up to 300 million euros in a year for Glybera.
Soliris
A course of treatment with Soliris can cost a patient up to $440,000 a year. Ecolizumab, a humanized (derived from mice) monoclonal antibody, was prescribed on the drug market under this brand name. In 2010, ecolizumab was named the most expensive drug on Earth. Soliris alleviates the suffering of patients with paroxysmal nocturnal hemoglobinuria. This is a very rare disease, the prevalence of which is 15.9 cases per million people. Only 8 thousand people on the planet have this diagnosis.
Paroxysmal nocturnal hemoglobinuria is a disease of the hematopoietic system, known since 1882 as life-threatening. The disease destroys red blood cells (erythrocytes) and manifests as anemia, bone marrow failure, abdominal pain, dysphagia, fatigue, chronic kidney disease and even. Soliris made it possible to raise the quality of life of patients with nocturnal hemoglobinuria to the population average level. However, ecolizumab does not reduce the chances of dying from a rare disease, making it possible to live longer without complications. The orphan drug was thought to help treat rheumatoid arthritis, which affects millions, but clinical trials were unsuccessful.
Elapraz
This orphan drug is used to treat Hunter disease (syndrome), or mucopolysaccharidosis type 2. This exceptionally rare recessive genetic disorder affects 2,000 people worldwide, of whom approximately 500 live in the United States. Hunter disease manifests itself externally as gargoilism, a characteristic rough appearance, as well as growth retardation and mental retardation.
The disease always resolves in acute form, making itself known when the patient is 2-4 years old. Because the disease is genetic cause, it is difficult to treat. The only drug designed to combat the cause of Hunter syndrome rather than the symptoms is Elaprase. The drug is a substitute for an enzyme, the deficiency of which the patient suffers from. It's called idursulfase and is a purified version of the human enzyme iduronate-2-sulfatase, approved for use after a successful clinical trial on 96 patients in 2006. Moreover, in a number of cases, after Elaprase infusions, patients developed life-threatening anaphylactic reactions. The drug for Hunter's disease costs $375,000 for a year, and one 3 ml dose costs about $3,400.
Naglazim
The drug Naglazyme (galsulfase) is used to treat mucopolysaccharidosis type 6 - polydystrophic dwarfism, Maroteaux-Lamy syndrome. This rare inherited disease is associated with a deficiency of the enzyme arylsulfatase. The first signs of the disease appear in the first year of a child’s life, subsequently the patient’s skeleton develops incorrectly, the mobility of the arms and legs is greatly reduced, the height of an adult in a severe case of the syndrome does not exceed 100 cm. All patients - and there are about 1,100 people on the planet - have problems with heart and nervous system, and about 1100 inhabitants of the planet suffer from the Maroteau-Lami syndrome.
Naglazyme, a drug to treat this type of mucopolysaccharidosis, costs more than $365,000 a year and is a laboratory-made modified human enzyme. It has been established that Naglazym solves many problems with skeletal development and joint mobility. At the same time, one of the most expensive medications in the world has a lot of side effects. Among them are hives, rash, swelling, pain in the chest and abdomen, fever.
Folotin
The drug Folotyn (pralatrexate) is used to treat a rare form of blood cancer called T-cell lymphoma.
Folotin is such an expensive drug that it is used only when nothing else helps, and the course intravenous treatment lasts 6 weeks and costs over $320,000. At the same time, Folotyn slightly prolongs the life of the patient. T cell lymphoma, which is why it is rarely used. Clinical trials of the drug are underway to determine its suitability for treating other types of malignant tumors blood cells and other types of cancer in general.
Aktar
Acthar is prescribed for the treatment of spasms and convulsions in young children (so-called infantile seizures, a rare form of epilepsy) aged 4 months to two years, as well as for several other ailments, including - multiple sclerosis, nephrotic syndrome and rheumatoid arthritis. Aktar is a specially purified corticotropin hormone of prolonged action in the form of a gel, used in the form of subcutaneous injections.
The drug Achtar is good for everyone, it has been known to medicine for a long time, but the price, nevertheless, reaches 200 thousand dollars per year (up to 26,000 for one bottle), and the income from the sale of the drug against infantile seizures is one hundred million dollars.
Myozyme
The drug Myozyme, judging by trade name, is an enzyme and is used to treat the rare, sometimes fatal Pompe disease. The disease is named after the Dutch pathologist who first described it in 1932. Pompe disease is hereditary. For a child to be born sick, both parents must have the mutant gene. It is believed that from 5 to 10 thousand people on Earth suffer from this disease. Dangerous symptom is muscle weakness, which can spread to the muscles of the heart and respiratory organs.
Myozyme is an alpha-glucosidase enzyme that breaks down glycogen, which is deficient in patients with Pompe disease. The drug allowed to reduce the number of patients requiring artificial ventilation lungs, and also increase survival. Doctors use Myozyme if the patient is under 8 years of age. The medicine is administered intravenously once every two weeks. The cost of a one-year course ranges from 100 to 300 thousand US dollars, and Myozim should be taken for life. Side effects include pneumonia and possible allergic reactions threatening the patient's life.
Cinriz and Firazir
Cinryze is one of the most expensive drugs on Earth. It is used to treat angioedema (HAE) - hereditary angioedema. It's rare genetic disease diagnosed in one person out of 50 thousand. With angioedema in children and adults, internal reactions occur in the blood, severe swelling of the arms, abdomen and larynx, face and genitals is observed during painful attacks - exacerbations of the disease.
Treatment of the disease - prevention of its attacks with the use of Cinryze is recommended to begin as early as possible, especially if the symptoms are life threatening strength. However, such treatment is not affordable for many patients - they will have to pay 350 thousand dollars a year for Cinriz. So expensive product is a human serum protein. In patients with angioedema, the liver cannot cope with the production of this protein. Cinriz is administered twice a week.
Another drug used to treat congenital angioedema is icatibant, a bradykinin receptor antagonist. One of the trade names of the drug is Firazyr. A package of three Firazira syringes costs more than 35 thousand US dollars.
Why are these drugs so expensive?
So-called orphan drugs from time to time become the heroes of sensational publications devoted to the high cost of such specific drugs. There are several reasons for this high cost.
Firstly, an amount of money of the same order is invested in the development of a new drug, be it orphan or mass market. The investment pays off through consumers. Roughly speaking, either a million patients will buy a drug for a dollar per dose, or one patient will buy a dose for a million.
Secondly, manufacturers of expensive drugs are confident that their products will be purchased at any price. Symptoms of many rare diseases so severe that there is no talk of refusing treatment.
Thirdly, there are no orphan drugs on the market. And finally, companies have to give away some of their expensive products to patients for free or sell them cheap.
Prices for biotech drugs for rare diseases continue to rise, flying into the stratosphere. There is no single price for the most expensive drugs in the world, and no one compiles a list of them. Therefore, Forbes, based on responses from surveyed Wall Street biotech analysts, obtained the proposed candidates for this list. We then surveyed manufacturers about the cost of a dose for a typical adult patient. Since the price value was not obtained, we used analysts' forecasts. And they found that prices vary depending on the dose, which varies depending on the patient's weight and other factors. We included medications on our list that cost $200,000 or more per year for the average patient. Some expensive cancer drugs that cost $200,000 for a year of treatment didn't make the list because the average patient only takes them for a few months. So this is the most expensive drugs in the world 2017.
9. Aldurazyme
Annual Cost: $200,000 Manufacturer: Genzyme, BioMarin
This drug treats Hurler syndrome (MPS I), which causes patients to lose moral and physical development at the age of 4 years. Six hundred people in the world suffer from it.
8. Tserezim
For Gaucher disease fat deposits accumulate in the spleen, liver, lungs, bone marrow, and sometimes in the brain, resulting in skeletal disorders and loss of lung and kidney function. The drug replaces an enzyme that patients with Gaucher disease lack. Worldwide, there are 5,200 patients with this disease.
7. Fabrazyme
Annual Cost: $200,000 Manufacturer: Genzyme
Fabry disease causes a burning sensation, purple spots, an enlarged heart, and kidney problems. Number of patients with this disease, exceeds 2,200 people worldwide. Seventh place among the most expensive drugs in the world 2015-2016.
6.Arcalyst
Annual cost: $250,000 Manufacturer: Regeneron
The drug treats Muckle-Wales syndrome, which affects 2,000 people worldwide. This syndrome causes recurring fevers, rashes, joint and kidney pain.
5.Myozyme
Annual Cost: $300,000 Manufacturers: Genzyme, BioMarin Pharmaceuticals
Myozyme replaces an enzyme that is missing in Pompe disease. Potentially fatal disease weakens muscles and enlarges the heart. No treatment for late stages patients are ultimately confined to wheelchairs and require mechanical ventilation. Nine hundred people worldwide suffer from this disease.
4. Cinryze
Annual cost: $350,000 Manufacturer: Viropharm
Cinryze treats hereditary angioedema (HAE) disorder immune system which causes swelling in abdominal cavity and lips The disease affects 6,000 people in the United States.
3. Naglazyme
Annual Cost: $365,000 Manufacturer: BioMarin Pharmaceuticals
Bronze in the ranking of the most expensive drugs. This medicine treats a metabolic disorder called Maroteaux-Lamy syndrome (MPS VI), which affects 1,100 people worldwide. Symptoms include an enlarged head, thick lips, and short stature. Patients may be chained to wheelchair from 15 years old.
2. Elaprase
Annual cost: $375,000 Manufacturer: Shire
This medicine treats something called Hunter syndrome (Mucopolysaccharidosis II, MPS II), which causes a flattened nose, enlarged tongue, difficulty breathing, and brain damage. There are two thousand patients in the world taking this medicine.
1. Soliris
Annual cost: $409,500 Manufacturer: Alexion Pharmaceuticals
The most expensive drug in the world. This drug belongs to paroxysmal nocturnal hemoglobinuria(PNG). It treats a disorder in which the immune system destroys red blood cells. Without treatment, as a rule, death occurs within 10 years. This disease affects 8,000 people in the United States.
The site administration, in turn, wishes you good health, all the best and so that you never need such expensive medicines.
In Germany last week they postponed the launch of a long-awaited drug on the market. we're talking about about the first medicine approved in Europe that can change the human genome. They postponed, as the commission’s decision says, “until confirmation therapeutic effect". Opponents radical intervention in human nature they celebrate victory. At the same time, a considerable part of the scientific community is convinced that humanity is needlessly slowing down progress, because when “editing” genes becomes an ordinary treatment, we will not only get rid of incurable diseases, but also radically improve the genome of humanity. Is the world ready for gene therapy, and is man ready for reasonable self-improvement, and if so, will there be a chance for new type treatment from Russians?
In a small ampoule with an inconspicuous sticker - absolutely clear liquid. This is Glybera - the most expensive medicine in the world. A course of treatment with this drug costs 1.1 million euros. One ampoule (53 thousand euros) contains hundreds of thousands of viruses into which scientists have inserted a copy of the working LPL gene (lipoprotein lipase gene) - it is responsible for the production of a special enzyme in the muscles that utilizes fat in our body. If the gene “breaks”, then the slightest amount fatty foods causes severe inflammation of the pancreas and blockage of blood vessels with plaques.
The mechanism of action is as follows: once in the body, “medicinal” viruses, according to their nature, are integrated into human DNA and replace the “broken” gene with the correct one. Opponents say differently: with just a few injections in the thigh, a person becomes a genetically modified organism (thus GMO), because his DNA includes artificial pieces of the gene. Be that as it may, the effect is that the body itself begins to produce enough enzymes to cope with fats, and the person gets a chance to live a full life.
The developers explain the fantastic cost by the fact that Glybera was developed for 25 years and it required huge investments. In addition, the circle of potential users of the drug is narrow: this gene disorder occurs in 1-2 people per million. It turns out this way: the cost of a course of treatment is comparable to the expensive drugs that such patients have to take all their lives to replace the missing enzyme.
However, the main precedent if this drug were to enter the European market would not be money. Following Glybera, dozens of gene-altering drugs could flood onto the EU market—all of which have been accumulating in the pantries of pharmaceutical companies for years. Judging by the fact that the European Commission (the EU government) and the European Medicines Agency (EMA) have given the green light, Europe is afraid of falling behind China in this area, where two gene therapy drugs have already been officially approved for sale (both oncology).
— The method that was used to deliver a copy of LPL into the genome is applicable to different drugs,” emphasizes Thomas Salmonson, Chairman of the Committee on medicines for humans at the EMA.—This means that we can talk about the treatment of many diseases caused by malfunctioning genes.
So why did the German commission (its official name G-BA - Joint Federal Committee) for the third time since 2012 refuses to release this gene therapy drug to the market? Official Clarification orders the developer company to provide “additional data on the therapeutic value of the drug,” but in fact it seems that officials are playing it safe: there were very loud scandals due to the fact that experimental gene drugs were killing people. Yes, today it is clear to everyone that these drugs are the future, but take responsibility for side risks officials controlling Europe's main pharmaceutical market again did not.
In conspiracy with the virus
The idea that by interfering with genes it is possible to rid humanity of previously incurable diseases has excited the minds of scientists since the mid-1970s, when basic methods had barely been developed genetic engineering. The experts immediately understood: The fundamental difference between the new method of treatment is that it does not eliminate the consequences of the disease , but its root cause. This process became more or less real after geneticists learned to correlate specific genes with diseases. After the “Human Genome” project (by deciphering the genome), it became clear: in fact, there are fewer genes responsible for the vital functions of the body than thought—“only” 20-25 thousand (and not 100 thousand). Wherein various kinds“breakdowns” in genes leading to malfunctions in the body are a common occurrence.
— In principle, in any population, every 150-160th child suffers from one or another hereditary disease, says the deputy director of the Medical Genetic Research Institute center of the Russian Academy of Medical Sciences Vera Izhevskaya.— For adults, these numbers are lower: one per 200-250 people. These diseases are not necessarily severe, because the genetic pathology varies. For example, there is a gene that causes significantly increased cholesterol. We also know that 50 to 70 percent of all congenital hearing loss and deafness are also caused by gene defects. And there are systemic situations when the heart, eyes, and intellect are affected.
The first serious attempts to “edit” the genome belonged to real luminaries of genetics, who chose as targets serious illnesses associated with a breakdown in a specific gene. The idea of genome “editing” was tested many times on plants and animals: a virus was chosen as a transport, which was “loaded” useful component. And so in 1999 James Wilson, director of the Gene Therapy Institute at the University of Pennsylvania, has begun testing a viral carrier for editing the human genome. Wilson was in a hurry: it would later turn out that he had violated the rules of clinical trials, in particular, he kept silent about the death of experimental monkeys.
An 18-year-old was given an injection with a drug to change the genome Jesse Gelsinger from Arizona. Jesse suffered rare disease: his liver did not produce the enzyme that breaks down ammonia - by-product vital activity of the body. Usually, pathologists die from this before reaching the age of five, but Jesse - a special case: the mutation was not inherited; it arose spontaneously in the body. After an injection containing “medicinal” viruses, he developed a fever and then developed multiorgan pathology. On the second day the young man died.
“Jesse underwent gene therapy based on adenoviruses,” explains Ancha Baranova, professor at the School of Systems Biology at GMU (USA), head of the Center for the Study of chronic diseases metabolism in the GMU College of Sciences, director of science at Atlas Biomedical Holdings, and a member of the American Society of Human Genetics.—They have now been abandoned because they cause severe immune reaction. It is impossible to find out whether it will happen or not until the adenovirus enters the body. We can say that Jesse's death was a tragedy not only for his parents, but for all gene therapy. Because immediately after it, all work related to the use of gene therapy on the human body was suspended.
Wilson's scientific career was undermined, and the Gene Therapy Institute was completely closed. An indefinite moratorium has been introduced in the United States on all experiments with “editing” the human genome. Scientists were indignant, but the moratorium was somewhat beneficial. We began to actively look for new ways to deliver gene drugs and settled on retroviruses. Unlike adenoviruses, they did not cause a disturbance in the immune system, but had another drawback - such viruses are integrated into the genome haphazardly, “where God sent it.”
In 2004, the French introduced a drug for the treatment of severe combined immunodeficiency, a disease in which a child is born without any immunity at all. All his life, unfortunately short, he lives in a sterile room and even communicates with his parents wearing rubber gloves. It was these children who were given a drug that corrected a defect in the gene. The effect was amazing - 10 children began to develop their own immunity, however, at the same time, two developed leukemia (they, fortunately, were cured). As it turned out, the retrovirus inserted a “therapeutic” gene next to the gene that causes this particular blood cancer. Gene therapy has once again been given the thumbs down: control over therapy research has become so strict that the flow of innovation has dried up for several years, both in Europe and the USA. Then China came out on top.
Made in China
“Almost immediately after these events, two drugs related to the delivery of healthy genes to the tumor were approved for use in China—Gendicine and Oncorine,” says Professor Ancha Baranova. “They are intended for treatment oncological diseases, more specifically, they activate the p53 gene, which is “responsible” for suppressing the growth of cancer cells.
It is interesting that the Chinese hastily brought to market not their own developments, but drugs that were made in South Korea and the USA, but did not reach the consumer due to legislative barriers. In the Celestial Empire they are not so scrupulous about the conditions of fulfillment clinical trials and, skeptics argue, are glossing over the true incidence of serious side effects. In other words, China chose to mass-produce an incompletely tested drug that would help a large number people (even though it may cause some unpredictable consequences). In Europe and the USA they took a different path: life potential victim side effects (even a single one) put more than hundreds of lives of potential patients, which, according to a number of experts, this drug could help.
While bioethics commissions are deciding who is right, over the past years the PRC has not only received substantial profits from the sale of drugs, but has also gained fame as the capital of gene therapy - people with severe forms of head and neck cancer come to Beijing from all over the world for treatment. This prompted a group of US researchers to write a letter to the leadership of the National Institutes of Health (which distributes the budget for biomedical science) in 2013, calling for them to abandon total control over gene therapy. After six months of deliberation, officials agreed with the scientists.
It is important to note that after the forced moratorium, many scientists abandoned the previous versions of transporter viruses. Today they give preference to the adeno-associated virus (AAV): the vast majority of people are its carriers, so it will not be met with hostility by the immune system. In addition, AAV has one great feature: different kinds viruses are embedded in certain organs- in the liver, eyes, brain cells and, as in the case of Glybera, mainly in the muscles. True, it also has disadvantages: it can “take away” a very small amount of medicinal material.
The second virus with which scientists are increasingly working is, oddly enough, the virus that causes AIDS. Researchers use its neutralized copy, which has promising properties from a therapeutic point of view: it can penetrate the cells of the immune system and does not activate oncogenes.
Awaken potential
Over the past few years, the situation with new “gene” drugs (all of which are undergoing one or another stage of testing) in the world has looked encouraging. Doctors have learned to suppress the development of complex leukemia using genes, restore vision to monkeys blind from birth and reverse muscular dystrophy. Moreover, in some cases, numerous injections are not necessary: for example, scientists packaged a gene that allows one to fight cystic fibrosis (a systemic hereditary disease characterized by damage to the exocrine glands and respiratory organs) in an aerosol for inhalation.
By the way, in our country in 2012 they issued registration certificate for its own gene therapy drug for the treatment of severe vascular lesions in the legs. The drug is a human VEGF 165 gene, which is responsible for the growth of new blood vessels. Thus, almost unnoticed by the world, we became the second country after China to approve gene therapy for use.
Of course, there are a lot of unresolved issues. For example, today we have learned to treat hemophilia (so far in animal experiments) by delivering a new copy to a small number of genes, which is responsible for the normal production of blood clotting factor. Compared to healthy body this amount is 10 percent, but this is quite enough so that, suppose, a person, having broken his arm, could wait for an ambulance and not die from blood loss. Another thing is those diseases when a gene needs to be replaced throughout the body, literally in every cell. Here science is still powerless.
“We must understand that the technology of integrating a working copy of a gene into the genome is not the only opportunity to achieve results,” reminds Professor Ancha Baranova, “sometimes it’s easier to somehow turn on a silent gene or activate a dormant one.” IN Lately this is done using double-stranded RNA. Imagine that in the future we will be able, over time, in older people to reduce the functioning of the gene responsible for the production of the very plaques in the brain that cause Alzheimer's disease. This opens up truly limitless possibilities for humanity.
The most expensive medicine was created in the USA, costing 850 thousand dollars per package. Remember this name: Luxturna. From now on it is the most expensive drug in the whole world. It will be produced by Philadelphia-based pharmaceutical company Spark Therapeutics, which specializes in gene therapy.
In December 2017, the US Food and Drug Administration (FDA) approved the gene therapy Luxturna for the treatment of the rare inherited retinal disease Leber amaurosis. Developed by the American company Spark Therapeutics, Luxturna became the first gene therapy that, using a genetically modified virus, corrects the genome of retinal and vision cells directly in the patient’s body.
The RPE65 gene encodes the production of normal vision enzyme and is responsible for the formation of light receptors in the eyes, but if a person gets a defective copy of it, it will cause Leber's amaurosis or retinitis pigmentosa. Both diseases result in vision impairment and the risk of blindness. Gene therapy could be the answer.
Luxturna is a solution with a genetically modified virus that carries the “correct” PRE65 gene. The virus is injected directly into eyeball and triggers production of the corrected enzyme in the retina. The operation takes only 45 minutes. It is reported that a single application of the drug is sufficient. The package will contain two doses - one in each eye (that is, $425 thousand per dose).
Testing has proven that Luxturna does indeed restore vision in those who have a rare form of inherited blindness. It is estimated that about two thousand people are forced to live with this diagnosis in the United States.
Initially, Spark Therapeutics was going to launch the drug at a price of a million dollars, but insurance companies convinced it to reduce this amount somewhat. In America, most prescription drugs are at least partially covered by health insurers.
And although this is a colossal price for the drug, Luxturna is a real breakthrough in medicine, because gene therapy can help fight both hereditary diseases, and with cancer, most cases of which begin with DNA damage, writes Bigpicture.
By far the most expensive drug in the world was the European-unlicensed gene therapy drug Glybera from uniQure. Its cost was $1.2 million. However, last year it was discontinued due to insufficient demand. This medicine is intended for gene therapy of a rare hereditary disease - lipoproteinase enzyme deficiency.
Expensive doesn't always mean effective. Indeed, some of the most expensive medicines give the least positive results. Cheaper to buy a house than some of these drugs. Of course, this list may change in the coming months…
Myozyme
Annual cost: $100,000 to $300,000Myozyme is a drug used to treat a rare but debilitating illness, glycogenosis (Pompe disease). It affects the skeletal muscles and heart of the patient.
Akthar
Annual cost: $300,000
Aktar is a drug used to treat seizures in children under two years of age. Since the FDA food products and drugs (FDA) has not approved Aktar, it is not only difficult to obtain, but also insurance companies, as a rule, do not cover the costs of its purchase.
All this leads to the establishment high prices and sales profits in excess of three-quarters of a billion dollars per year.
Folotyn
Annual cost: $320,000
Folotin is used to fight a rare and aggressive type of cancer, T-cell lymphoma. For the patient, a six-week course of treatment with Folotin is the last chance to stop the disease. Although the drug generates more than $50 million in revenue annually, the drug has not been shown to significantly prolong life, making it an extremely expensive last resort.
Cinryze
Annual cost: $350,000
This drug is used to treat angioedema- a hereditary disease that affects one in 50 thousand people and causes swelling of the hands, throat and abdominal cavity. Produced from human blood, Cinryze has shown to be quite effective in the fight against this disease.
Naglazyme
Annual cost: $365,000
Used to treat a childhood disease known as Maroteaux-Lamy syndrome, which affects connective tissues. People suffering from this syndrome have abnormally developed muscles, joints and tissues, which often results in dwarfism.
Children with this disease also suffer from neurological and heart diseases, visual and hearing impairments, and brain damage. Neglazyme helps stimulate joint mobility and tissue growth.
Elaprase
Annual cost: $375,000
The drug, which costs more than most homes, helps fight Hunter syndrome, a rare inherited disorder that impedes physical growth and brain development.
Vimizim
Annual cost: $380,000
Vimizim is an enzyme used to treat Morquio syndrome. This disease prevents the breakdown of long-chain sugar molecules in the patient's body, leading to disturbances in the heart and skeletal development, dwarfism and other serious defects.
Soliris
Annual cost: $440,000
Soliris regularly occupies a leading position in the lists of the most expensive drugs, but at least it can also claim the title of very effective means to treat paroxysmal nocturnal hemoglobinuria, which affects about eight thousand Americans.
The disease destroys red blood cells, making the patient more susceptible to infection, anemia, and blood clots. Soliris helps to reduce these manifestations by 90%. This drug has also proven useful in treating atypical hemolytic uremic syndrome, another rare and sometimes fatal disease.
Glybera
Annual cost: $1.21 million.
And the title of the most expensive drug in the world goes to Glybera, a drug that has not yet been approved in the United States, but was recently approved in the EU. Glybera is a gene therapy used to treat familial lipoprotein lipase deficiency, a disease that affects one in a million people. In EU countries, less than 200 people suffer from it. Deficiency causes extremely painful swelling of the pancreas.
