The most expensive therapies and drugs in the world

These therapies and medications are sold at very high prices. In fact, their prices are so high that they challenge not only patients but also the health care system and insurance companies, according to Scientific American magazine.

Skysona is a gene therapy with a one-time treatment cost of $3 million that slows the progression of adrenoleukodystrophy (CALD).

The most expensive therapies and medications in the world include:

Skysona

Skysona is a breakthrough gene therapy with a one-time treatment cost of 3 million USD, about 73 billion VND. This therapy was developed by the biotechnology company Bluebird Bio (USA), which has the effect of slowing the progression of adrenoleukodystrophy (CALD).

CALD is a rare genetic disorder that affects the nervous system. The Skysona therapy involves modifying the patient’s stem cells to include the ALDP gene. This slows the progression of the disease and improves the quality of life for those with the condition. The therapy is expensive because of the research, development, and manufacturing costs.

Zynteglo

Zynteglo is a lentiviral vector gene therapy and is also developed by Bluebird Bio. The cost of this therapy is up to 2.8 million USD, about 68 billion VND, for one treatment. The subjects of the drug are patients with transfusion-dependent thalassemia (TDT). This is a rare blood disorder that requires patients to have regular and lifelong blood transfusions to survive.

Zynteglo therapy will reduce or even eliminate the need for blood transfusions, thereby improving quality of life as well as the physical and financial burden of blood transfusions.

Zolgensma

Zolgensma is considered a revolutionary gene therapy to treat spinal muscular atrophy (SMA), a rare genetic disease in children. The drug was developed by the Swiss pharmaceutical company Novartis and was approved by the US Food and Drug Administration (FDA) in May 2019.

The average cost of this treatment is $2.1 million, about VND50 billion. Zolgensma works by replacing the defective or missing gene in survival motor neuron 1 (SMN1) cells. It is this abnormality that causes the disease.

Zokinvy

Zokinvy is used to treat Hutchinson-Gilford progeria syndrome (HGPS) and Laminopathies progeroid syndrome. These are extremely rare genetic diseases that cause premature aging in children.

Zokinvy was developed by the biotechnology company Eiger BioPharmaceuticals (USA). The cost of treatment with this therapy is 1.7 million USD/year, about 25 billion VND. The drug is taken orally, has the effect of delaying the progression of the disease and improving the quality of life for children, according to Scientific American.

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