New York: A company has developed a successful therapy for the rarest genetic blood disorder, but it has been described as the costliest treatment in human history.
The company claims that although this amount is high, the rare disease costs even more over a lifetime if there is no therapy.
It's a cell-level gene therapy that's the first for people with a rare blood disorder and has been mandated by the US Food and Drug Administration. However, a one-time treatment costs $2.8 million, making it the most expensive treatment.
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In this, stem cells are taken from the patient's blood marrow and some are genetically modified to make multiple copies of the correct gene that makes active red blood cells. Now these modified cells are injected into the human body. A one-time treatment is enough for a lifetime.
When it was tested on some patients, 89% of the patients recovered without needing a repeat blood transfusion and had no adverse effects. While other similar gene therapies often increase the risk of cancer.
However, even after 15 years of treatment, these patients were seen by an independent panel of experts, but some patients were also found to be at risk of cancer. But due to its overall benefits, it has been accepted by the FDA for treatment.
The approval of Zantiglo is a landmark case as it is the third gene therapy that has successfully crossed over to consumers. Despite all this, its price is reasonable and has earned the title of the most expensive therapy in the United States. Earlier in 2019, the price of Zolgensma, a gene therapy, was estimated to be around 2 million US dollars.
It has been developed by a company called Bluebird Bio which can protect beta-thalassemia patients for life. But if the patient continues his traditional treatment throughout his life, he can reach up to 6.4 million dollars. That's why the company has described it as cheap as only a lump sum has to be paid by the patient.
But the patients of this rare disease are very few, only 1300 to 1500 people suffer from it in America.
