The five most expensive drugs in the world – the record holder costs over 2 million dollars!

Proven content

New records are breaking on the drug market, and most often they concern drugs used in the treatment of rare diseases, the so-called orphan drugs. The following five will make more than one patient dizzy …

Zolgensma Therapy

Information about the approval of the new most expensive drug in the world by the American Food and Drug Administration (FDA) reached us in May this year. Treatment costs are $ 2,1 million, or € 1,87 millionwhich makes the medicine used in the form of a single infusion the most expensive medicine in the world. Zolgensma gene therapy is a chance for children under two years of age suffering from SMA, i.e. spinal muscle atrophy. The disease often leads to paralysis, breathing difficulties, premature death, or lifelong disability.

Zolgensma therapy involves replacing the damaged SMN1 gene with a functioning copy that is administered to patients. This is done as a single infusion and may stop the disease from progressing. However, Novartis’ medicine cannot remove the damage that has already been done to patients. It is therefore of paramount importance to identify the symptoms of spinal muscular atrophy as soon as possible.

Until now, only the drug Spinraza, manufactured by the US biotechnology company Biogen and costing hundreds of thousands of dollars per year, has been used to treat spinal muscular atrophy.

1. Eight drugs that are better not to be combined with alcohol. Be careful!

Glybera

It is poorly used in gene therapy for the treatment of lipoprotein lipase deficiency. It is a very rare disease (200 people in Europe suffer from it) associated with the inability to properly digest fats. The excess of undegraded fats causes severe pain in the abdominal cavity and damages the pancreas. The drug lowers their concentrations and prevents inflammation of this very important organ, which is dangerous to health and life. Glybera was developed by uniQure from Amsterdam and in 2012 it gained five-year approval in the European Union.

Her single dose cost € 1 million. However, in five years, only one patient chose to undergo this expensive treatment. As there was no chance of selling larger quantities of the drug, the company did not apply for re-registration. Glybera was available in the EU until October 25, 2017. The drug has not been registered in the USA.

1. 22q11.2 deletion syndrome. A defect with which one in two to four thousand is born. kids

Spinraza

In the United States, it was approved for SMA treatment in December 2016 and in the European Union in June 2017, becoming the first approved treatment for spinal muscular atrophy. The cost of one dose in the EU is about 90. euro, while the cost of one-year therapy in the first year of treatment is approximately PLN 540. euro (6 injections) and 270 thousand. euro in subsequent years of treatment (3 doses of the preparation). Spinraza is a drug that works at the gene level.

It modifies the process of DNA folding proteins so that the protein created by the stock SMN2 gene is more stable and can effectively replace the one lacking in SMA patients. It allows the motor nerves that transmit signals from the brain and spinal cord to the muscles to function efficiently. The drug is administered directly to where it is needed most – into the spinal canal – by intrathecal injection.

Luxturna

Intended for the treatment of patients with vision loss due to confirmed biallelic hereditary RPE65-dependent retinal disease. Patients with IRD (hereditary retinal disease) due to biallelic mutations in the RPE65 gene often experience night blindness due to reduced sensitivity to light in childhood or adolescence, as well as involuntary forward and backward movements of the eyeballs. As the condition progresses, peripheral vision worsens and tunnel vision develops, eventually leading to complete blindness. The efficiency and independence of patients is severely limited.

Luxturna is intended for adults and children who have inherited the mutation from both parents and who have a sufficient number of viable retinal cells. A single injection of the drug delivers the RPE65 gene to the cells of the retina restoring the production of the required enzyme that improves the patient’s ability to detect light. Drug therapy is very expensive – one eye treatment costs $ 425, both 850.

1. Medications that are stored wrongly are poisonous

Raptured

Ravicti is a medicine intended for use in urea cycle disorders in adults and children over two months of age, when the diseases cannot be treated solely by dietary changes. Patients with urea cycle disorders are unable to remove nitrogen from the body due to the lack of certain liver enzymes. In the body, nitrogen is converted into ammonia, the accumulation of which is harmful.

Ravicti is used in patients lacking one or more of the following enzymes: carbamoylphosphate synthase I, ornithine carbamoyltransferase, argininesuccinate synthetase, argininesuccinate lyase, arginase I and ornithine translocator. The drug contains the active substance glycerol phenylbutyrate.

Because the number of patients with urea cycle disorders is low, they are considered rare, and Ravicti was designated an ‘orphan medicine’ on 10 June 2010. The cost of the annual treatment is almost PLN 794 thousand. dollars.

Read also:

1. These medications can cause ulcers. Be careful!
2. Seven foods that harm the intestines. Watch out for them!
3. Natural remedies for a headache. Try before you reach for medications

You haven’t been able to find the cause of your ailments for a long time? Do you want to tell us your story or draw attention to a common health problem? Write to the address [email protected] #Together we can do more