Virus for 850 thousand dollars: the most expensive medicine in the world was released in the USA

The most expensive medicine in the history of the United States goes on sale — at a cost of 850 thousand dollars (48 million rubles) per package. Remember this name: Luxturna. From now on, it is the most expensive drug in the whole world. It will be produced from the beginning of this year by the Philadelphia-based pharmaceutical company Spark Therapeutics, which specializes in gene therapy.

Source: CBS.com

In December 2017, the U.S. Food and Drug Administration (FDA) approved Luxturna gene therapy for the treatment of a rare hereditary retinal disease — Leber's amaurosis. Developed by the American company Spark Therapeutics, Luxturna became the first gene therapy that uses a genetically modified virus to correct the genome of retinal cells directly in the patient's body, providing partial restoration of vision.

The RPE65 gene encodes the production of an enzyme necessary for normal vision and is responsible for the formation of eye light receptors, but if a person gets a defective copy of it, it will cause Leber's amaurosis or retinitis pigmentosa. Both diseases are expressed in the deterioration of vision and the risk of blindness. Gene therapy can be a salvation.

Luxturna is a solution with a genetically modified virus carrying the "correct" PRE65 gene. The virus is injected directly into the eyeball and triggers the production of a corrected enzyme in the retina. The operation lasts only 45 minutes. It is reported that a single application of the drug is sufficient. The package will contain two doses — one in each eye (that is, 425 thousand dollars per dose).

Testing has proven that Luxturna really restores vision in those who have a rare form of inherited blindness. It is estimated that about two thousand people are forced to live with this diagnosis in the USA.

Initially, Spark Therapeutics was going to release the drug to the market at a price of a million dollars, but insurance companies convinced her to reduce this amount somewhat. In America, most of the drugs prescribed by a doctor are at least partially paid for by health insurance companies.

And although this is a colossal price for the drug, Luxturna is a real breakthrough in medicine, because gene therapy can help in the fight against both hereditary diseases and cancer, most cases of which begin with DNA damage.

To date, the most expensive drug in the world was the gene therapy drug Glybera from UniQure, which was used in Europe, but did not have a license from the United States. Its cost was $1.2 million. However, last year it was discontinued due to insufficient demand. This medicine is intended for gene therapy of a rare hereditary disease — deficiency of the enzyme lipoproteinase.