USA- The Food and Drug Administration has validated a tailored gene therapy as a potent new treatment for people with the genetic blood disorder beta-thalassemia.
Created by the biotechnology company Bluebird bio, the therapy, which in clinical trials demonstrated a significant effect, will be one of the priciest drugs available, costing US$2.8 million per patient.
The condition beta thalassemia, which in extreme cases necessitates ongoing blood transfusions for life, has long been identified as a potential target for gene therapy.
The first of numerous treatments currently in development to be commercially available in the United States is Bluebird’s Zynteglo, which will be sold to patients as an alternative to those transfusions and the adverse effects that come with them.
The approval of Zynteglo is particularly important for Bluebird, a pioneer in gene therapy that has recently struggled and is now in threat of going bankrupt.
Zynteglo and other gene therapies Bluebird is researching, have taken more than a decade of study to get to this point and doing so have cost the business billions of dollars.
Before approving Zynteglo, the FDA has had to balance the treatment’s obvious advantages against worries that it might lead to cancer.
In 2021, the Massachusetts-based company removed gene therapy from the German market after failing to reach an agreement with German health authorities on the treatment’s price.
This decision was a sign of the difficulty biotech companies face when attempting to market genetic medicines as extremely expensive one-time treatments.
In part, as a result, the biotech company revealed intentions to lay off employees and reorganize its operations in Europe to concentrate on “priority” areas.
The company held negotiating price talks with additional European nations, and it hoped to provide updates on those discussions later in the year.
Zynteglo is created from the stem cells of each patient. It is altered when cells are produced in a lab oxygen-carrying protein hemoglobin, which is misformed in beta-thalassemia and sickle cell disease.
A manufactured virus that integrates into the cell genome is used by scientists to add the gene. Since gene therapy research frequently employs such viruses, earlier that year’s Bluebird cancer findings sparked broader concerns about the development of the sector.
Before approving Zynteglo, the FDA has had to balance the treatment’s obvious advantages against worries that it might lead to cancer.
Zynteglo trials have not revealed any occurrences of cancer, however, a research participant in a study of a different Bluebird gene treatment for sickle cell disease, acquired a kind of leukemia.
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