Casgevy: First-ever CRISPR-based medicine gets FDA nod for beta-thalassemia treatment

Representation for DNA
Representation for DNA

United States: The Food and Drug Administration (FDA) of the United States has given a green flag to a medication to treat sickle cell disease – Casgevy, to treat another blood disorder named beta-thalassemia. To highlight, the sickle cell treatment was approved by the FDA in December 2023.

Casgevy – a CRISPR-based medicine!

Casgevy is the first-ever CRISPR-based medicine approved for usage in the United States. The medicine allows gene editing for the development of the treatment. The experts have mentioned that just a single dose of medicine could permanently change the DNA of the blood cells of the patient.

Representation for Casgevy | Credits: Google Images

However, a few experts are concerned and highlighted that the treatment and relief would not come cheap as a single dose of Casgevy costs US$ 2.2 million for both its uses – as sickle cell treatment and beta-thalassemia, according to the reports published by CNN.

What is beta-thalassemia?

Beta-thalassemia is a blood disorder in which the human body fails to make enough oxygen-carrying molecules in blood. The molecules are scientifically known as hemoglobin.

A beta-thalassemic infected person can become anemic, can feel weak and tired, and can also experience breath shortness. The infection can shorten the life span of a person.

Approval to Casgevy – an important step towards advancement!

Various health experts have mentioned that after this latest approval, Casgevy will be given to patients aged 12 and above. According to health experts, the dose will be given with ‘transfusion-dependent beta-thalassemia.’

The approval was addressed by the director of the Office of Therapeutic Products within the FDA’s Centers for Biologics Evaluation and Research – Dr. Nicole Verdun, “Today’s approval is an important step in the advancement of an additional treatment option for individuals with beta-thalassemia, a debilitating disease that places individuals at risk of many serious health problems,” as per HealthDay News.

Representation for DNA transfusion | Credits: Google

In a news release, she further explained, “The approval of a cell-based gene therapy for this condition using CRISPR/Cas9 technology reflects FDA’s continued commitment to supporting safe and effective treatments that leverage the most promising and cutting-edge medical technologies.”

Casgevy’s approval in December

Casgevy attained FDA approval in the month of December. The medicine was approved to treat sickle cell disease. At that time, Verdun stated, “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”

According to reports by CNN, the decision was supposed to come two months later but was made earlier.

How Casgevy works?

The medication is made with the help of the stem cells of the infected person, which is initially removed and then modified using a gene-editing technique known as CRISPR/Cas9. Following this, the cells are transplanted into the body of the patient. This transplantation increases the production of hemoglobin – easing the symptoms of the disease.

Certain side-effects of medication:

As per the Food and Drug Administration, some of the common side-effects of the treatment are fever because of the lower number of white blood cells and decreased appetite.

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