In the United States released the most expensive drug — Luxturna

Initially, the company was going to sell it for 1 million dollars per dose. Insurance companies have achieved a price reduction of 15 percent. In America, part of the cost of prescription drugs is paid for by insurance. But all the same — you can not call the drug available. So Luxturna is the most expensive medicine in the US and around the world. It will be produced by Spark Therapeutics company starting this year.

The drug is designed to fight the hereditary disease of the eye retina — amaurosis Leber. This disease often provokes blindness. Light-sensitive cells die in the retina. Usually, the disease is found in the first months of life. Visually this is imperceptible, however, without causing pathological changes in the eye, the disease can take away the sight completely. If this did not happen immediately, by the age of 10 the child still, most likely, will go blind.

In the human body there is the gene RPE65. It encodes the production of an enzyme, which is necessary for the normal functioning of vision. In addition, he is responsible for the formation of light receptors in the eyes. But it happens that a person has a defective copy of this gene. In such cases, it causes Leber’s amaurosis or pigment retinitis.

Gene therapy can help. The most expensive drug, Luxturna, is based on a solution with a genetically modified virus that carries the necessary gene. Recently, medicine has moved far and now even genetic engineering has become possible. A genetically modified herpes virus will help in the fight against cancer. And now another virus is created, which is introduced directly into the retina of the eye and starts the production of the necessary enzyme. Directly in the human body is corrected by the genome of the retina, which leads to a partial restoration of vision. The whole operation lasts only 45 minutes. It is enough only once to carry out the procedure. Each eye is administered one dose of the drug. Each costs 425 thousand dollars.

The US Food and Drug Administration (FDA) has already approved gene therapy for the most expensive drug.

The tests were successful — Luxturna does restore vision in patients suffering from a rare form of hereditary blindness. According to statistics, about 2000 people in the United States suffer from this disease.

Although Luxturna is the most expensive medicine, it still has no analogues, and is a breakthrough in gene therapy. This technology can be used in the fight against cancer, hereditary diseases.