World’s most expensive drug approved by FDA. The therapy costs more than two million dollars

Spinal muscular atrophy (SMA) primarily affects children. The first symptoms can be seen in the first months of life. This is the so-called. The infantile form of this disease, whichora has the worst prognosis. Depending on the timing of the wyrotion is also an intermediate form, when the first symptoms occur between 6. a 18. month of life and the juvenile form, when symptoms appear after 12. month of life. The adult form is also very rare, when it affects individualsob around the age of 40. It is the mildest form of the condition.

SMA is an inherited disease linked to a defective SMN1 gene. A defective gene thatory causes spinal muscular atrophy, prevents the body from producing enough protein, whichore allows the nerves that control movement to work normally. Nerves due to lack of adequate protein die off.

The condition weakens children’s muscles to such an extent that they become unable to move and eventually are unable to swallow or breathe. In the most common type of the disease, ktory is roalso the most severe, dying at least 90 percent of the. small patientsow. Children with less severe types become disabled more slowly and can live up to several decades. The disease is diagnosed on average once in every 10,000. birth.

Zolgensma gene therapy approved by the FDA (Food and Drug Administrationow), ktoThe tasks are focused on m.in. wokoł food control, the drugoin or medical devices marketed in the US, targets the defective SMN1 gene. It has been approved for use in children under the age of 2, in ktorych SMA has been genetically confirmed. The therapy is a one-time infusion, ktora lasts about an hour.

The drug is supplied to the market by Swiss manufacturer Novartis. One infusion costs 2 million 125 thousand dollarsow. Because of this high costow manufacturer offers payment in five installments. In addition, if the drug does not work, the company has pledged to provide a rebate. The price of the treatment means that without state aid, few will be able to afford the treatment.

Zolgensma therapy works by delivering the correct copy of the defective gene, whichory enables the comohe nerve cells to start producing the needed protein. This stops the degeneration of the comonerve rec and allows the child to develop normally.

In clinical trials, children with the most severe form of the disease, whichore received the infusion within 6 months of birth, they had at least limited muscle problems, but no longer lost muscle control. However, the use of the drug does not undo the damage already doneod, which is why early diagnosis and rapid administration of the correct copy of the gene is important in therapy.

Children whoore taking part in the study are now 4-5 years old and show no signs ofoin the disease. However, on this basis, it cannot be ruled with certainty that a single infusion stops the disease for life.

An alternative to Zolgensma is a drug called Spinraza, ktory roIt is also approved for use in the U.S. In this case, therapy looks different. Instead of a single treatment, it should be administered every four months. Biogen, the manufacturer of the Spinraza drug, collects 750,000. dollaroin the first year, and then 350,000. annually.