The U.S. Food and Drug Administration has approved Zinteglo for gene therapy for one of the rarer types of anemia in Bluebird bio. This has been reported Reuters.
According to Bluebird Bio, there are approximately 1,500 beta thalassemia patients in the United States who can begin treatment with Zynteglo by the end of 2022. One shot would cost $2.8 million, enough to completely eliminate symptoms. True, it will be done before the 70-90 days required to prepare the drug before the injections.
Beta thalassemia is an inherited disease caused by several different mutations that reduce hemoglobin production in red blood cells.
Zinteglo is a drug for gene therapy. Its use includes removing bone marrow cells and introducing the “correct” version of the gene responsible for beta thalassemia formation. The cells are then injected back into the patient’s bone marrow. In 32 of the 36 patients who participated in the study, the cells began to produce normal amounts of hemoglobin, and the patients became completely independent of blood transfusions.
The FDA has warned of the potential risk of blood cancer associated with therapy use. But during clinical trials, such cases, among other serious side effects, were not observed. Despite the high cost of treatment, it may be more profitable than frequent lifetime blood transfusions, which the company estimates costs $6.4 million.
In the EU, the drug was approved in 2019, even before clinical trials were completed. Zynteglo’s cost in the European market is much lower: $1.77 million per pack. Treatment, as a rule, is paid not by the patient himself, but by the insurance company or the state. Previously, the record was held by Novartis’s Zolgensma ($2.1 million per pack), which is designed to halt the progression of spinal muscular atrophy.