United States: The first ever CRISPR-based medicine, Casgevy, has been approved for a second use by the US Food and Drug Administration (FDA).
In December, Casgevy received its first approval to treat sickle cell disease.
Now, the novel treatment can also be used for the treatment of transfusion-dependent beta-thalassemia among people 12 and older.
According to CNN Health, beta thalassemia is also an inherited blood disorder, just like sickle cell disease.
The decision came two months earlier than the FDA’s deadline to make the decision, which is also called the PDUFA date.
More about Casgevy
The process of making Casgevy involves a genetically modified version of a person’s stem cells made with the help of a precision gene editing technique called CRISPR/Cas9.
The newly modified cells are then transferred back into the body. There, they grow and multiply and hence increase the count of hemoglobin. The overall effect leads to a decrease in symptoms of the diseases, CNN Health reported.
Estimated cost of the treatment: Experts
For both sickle cell disease and beta-thalassemia, the treatment is marked for $2.2 million.
Dr. Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, told in a news release, “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,” reported CNN Health.
About Beta thalassemia
It is a body condition in which enough production of hemoglobin doesn’t take place. Hemoglobin is the oxygen-carrying molecule in the bloodstream.
It results in feeling weak due to the development of anemic conditions. It also leads to making the person feel tired and out of breath.
Moreover, beta thalassemia has the tendency to shorten a person’s life span, as reported by CNN Health.
What treatments were available in the past?
With frequent blood transfusions, the patient managed to control the disease. However, repeated transfusions also can lead to another problem called iron overload syndrome.
In order to cure the added problem, other treatments also need to be administered!
In the US, there are almost 1,200 patients suffering from thalassemia. The level of severity is extreme enough to be recommended to go through blood transfusions, as stated by the Boston Children’s Hospital.
About CRISPR
CRISPR stands for clustered, regularly interspaced short palindromic repeats, seen as a milestone discovery in the medical field. With the help of CRISPR technology, exact changes can be made in DNA to cure any number of disorders.
Discovered by Emmanuelle Charpentier and Jennifer Doudna, won the Nobel Prize for discovering the CRISPR tool.
As per the Noble Prize website, the technology has “revolutionized the molecular life sciences, brought new opportunities for plant breeding, is contributing to innovative cancer therapies, and may make the dream of curing inherited diseases come true,” report by forbes.com.