Experimental medicines give hope to people suffering from serious illnesses. Pharmaceutical companies have to spend a lot of money on drug development. Therefore, the price of medicines is high.
Sebastian is a 10-year-old boy who suffers from Duchenne muscular dystrophy. This disease damages muscles and leads to fatal outcomes. A new gene therapy could help, but it costs 2.5 million euros.
Questions have arisen about the drug Elevidys. The European Medicines Agency has halted its registration. Three patients have died after using this drug. One of the causes of death was liver damage.
Drug development studies did not show that the drug helps patients improve their mobility. The mechanism of action of the drug could not be proven.
The US Food and Drug Administration has approved the drug. The US considered the expression of truncated dystrophin in muscle cells when evaluating the drug.
The price of medicines is determined by several factors. Drug development is a long and expensive process. Companies want to make a profit. Medicines for rare diseases are expensive because they are used by few people.
Elevidys is a gene therapy drug. It uses a virus that helps produce truncated dystrophin. This protein helps muscle cells function normally. The drug is administered once and its effect can last for several years.