FDA clears first CRISPR treatment for a second disease, beta thalassemia

By Brenda Goodman, CNN

(CNN) — The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease.

The groundbreaking treatment can now also be used to treat transfusion-dependent beta thalassemia in people 12 and older. Like sickle cell, beta thalassemia is an inherited blood disorder.

The FDA’s decision was expected, but it comes about two months earlier than the agency’s deadline for acting, called the PDUFA date.

To make Casgevy, a person’s stem cells are genetically modified using a precision gene editing technique called CRISPR/Cas9.  The modified cells are then transplanted back into the body, where they grow and multiply and increase the production of hemoglobin, which decreases symptoms.

The treatment lists for $2.2 million for both sickle cell disease and beta thalassemia.

“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,” said Dr. Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, in a news release.

Beta thalassemia is a condition in which the body doesn’t make enough hemoglobin, the oxygen-carrying molecule in blood. It causes a person to be anemic, which makes them to feel weak, tired and out of breath. Beta thalassemia can shorten the duration of a person’s life.

In the past, it has been managed with frequent blood transfusions. Repeated transfusions can lead to another problem called iron overload syndrome, which must then be managed with other treatments.

There are at least 1,200 people in the United States with thalassemia severe enough that it must be managed with blood transfusions, according to Boston Children’s Hospital.

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