10 most expensive drugs in the world
• Even an absolutely healthy (or, rather, underexamined) person is forced to take medicine from time to time to relieve a headache or toothache. While most drugs are expensive, you don't have to take out a loan or sell a kidney to buy them.
• However, the cost of some drugs can discourage not only the average Russian, but also a very rich person. We present you the top 10 most expensive drugs in the world.
• Ilaris is an injectable immunosuppressant approved for the treatment of acute gouty arthritis, systemic juvenile idiopathic arthritis in young children, and cryopyrin-associated periodic syndrome (CAPS), a group of rare diseases caused by mutations in certain genes.
• People with CAPS often have bouts of fever and inflammation without the usual causes, such as infections or viruses.
• Patients with pheochromocytoma, a hormonally active tumor of the adrenal glands, produce excessive amounts of norepinephrine and epinephrine, which raise blood pressure. To prevent this condition, patients with pheochromocytoma are forced to take a drug called Demser.
• It is also used for the long-term treatment of symptoms associated with a tumor.
• It is the drug of last resort for those with relapsed or refractory peripheral T-cell lymphoma, a rare and highly aggressive cancer. It is used only if other methods of treatment have failed.
• At the same time, even the use of Folotin does not guarantee an improvement in survival without progression of the disease, or at least a general extension of the patient's life expectancy.
• This medicine is used to treat genetic diseases – one of the most expensive groups of diseases. Soliris can be prescribed in case of:
- a rare and life-threatening chronic blood disorder called Atypical Hemolytic Uremic Syndrome. It affects both children and adults, and leads to the constant activation of the part of the immune system responsible for the destruction of infectious agents and the removal of dead cells. Simply put, the immune system begins to attack the body's own, healthy tissues.
- Paroxysmal nocturnal hemoglobinuria. The rarity of this condition is evidenced by the fact that for every million people there are no more than 15,9 cases of PNH. In patients, blood cells begin to die during sleep. Why – no one knows.
• At one time, Soliris was considered the most expensive drug in the world, but now there are drugs that compared to which Soliris is given almost for nothing.
• In 2019, Russian media reported that the pharmaceutical company Generium had registered the Russian equivalent of Soliris (eculizumab). It's called Elizaria and will cost a quarter less than the original. All components of the medicine, including the production of the substance for it, are domestic. This is the most expensive drug.
• One of the most expensive medicines in the world is used to treat Pompe disease, in which a person's glycogen breakdown processes in nerve and muscle cells are disrupted.
• This is due to the fact that the patient's body lacks an enzyme called "acid α-1,4-glucosidase." And Lumisim (aka (alglucosidase alpha) replaces the missing enzyme.
• Without treatment, Pompe disease leads to death from heart or respiratory failure in children. If the disease occurs in adulthood, a person slowly loses his sight due to myopia, and he has liver damage.
• Brineira is a super-expensive medicine that is used in the treatment of late childhood Batten disease (aka Bilshevsky-Jansky disease). It is quite rare, about 1 person in 200. With this hereditary pathology, vision quickly deteriorates up to complete blindness, there are often convulsions that are not relieved by medication, and the patient's motor activity is seriously impaired.
• And although Brineira does not provide a complete cure, it slows down the onset of disability and alleviates the symptoms of the disease in young patients 3 years of age and older.
• Loss of vision is one of the worst things that can happen to a person. The prospect of being forever in the dark is not as daunting as having to part with $850. Unless, of course, you have them.
• The developer of the drug, Spark Therapeutics, originally planned to sell its development for $1 million. But then she reduced her appetite at the insistence of insurance companies.
• The miracle drug, whose principle of action is based on gene therapy, does not restore sight to everyone, but only to those who are affected by a rare inherited form of blindness. For example, in the United States, about 2000 people suffer from this.
• To cure blindness, a single injection of Luxturna in each eye is enough (425 thousand dollars per injection, respectively). This novelty compares favorably with other drugs that need to be taken for a long time, or even a lifetime.
• Since June, Ravikti has received the status of “medicine for orphans” (a drug used for rare diseases).
• Ravikti is used to treat patients with urea cycle disorders. And although the name of the disease sounds frivolous, there is a great danger behind it.
- Patients are deficient in some liver enzymes, making the body unable to get rid of nitrogen waste.
- As a result, toxic ammonia begins to accumulate in the blood of adults and children.
- And this can lead to permanent brain damage, coma and death.
• The human liver has an enzyme with the long name N-Acetylglutamate Synthase. It is responsible for the processing of excess nitrogen, which is formed during the metabolism of proteins. And with hyperammonemia, this enzyme is not enough, and ammonia, aka nitrogen hydride, quickly accumulates in the blood. This leads to coma and subsequent death.
• This is a dangerous condition and prevents the drug Karbaglu. And given that the lack of the enzyme is a lifelong condition, you will have to take this drug for as long as the patient lives.
• This drug received a green light in the US as recently as May for the treatment of pediatric spinal muscular atrophy. Zolgensma replaces the mutated "defective" gene with a functional one.
• For a complete cure for the disease, just one intravenous injection is enough, but it is fantastically expensive. And the tool is focused on a very small group of people, because around the world, about 2500-3500 children suffer from spinal muscular atrophy. This disease progresses rapidly, and by the age of two, sick children who do not receive the necessary treatment usually die of respiratory failure.