FDA approves new gene-editing treatment for sickle cell disease

The U.S. Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, an illness that affects more than 100,000 Americans.

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The therapy, called Casgevy, is the first treatment to be approved in the United States that uses the gene-editing tool CRISPR.

“I think this is a pivotal moment in the field,” said Dr. Alexis Thompson, chief of the division of hematology at Children’s Hospital of Philadelphia, who has previously consulted for Vertex Pharmaceuticals, the company that makes the new treatment.

“It’s been really remarkable how quickly we went from the actual discovery of CRISPR, the awarding of a Nobel Prize, and now actually seeing it being an approved product.”

Casgevy works by editing the DNA found in a patient’s stem cells to remove the gene that causes sickle cell.

Until now, the only known cure for sickle cell disease was a bone marrow transplant from a donor. The procedure has many risks involved, including that the person’s immune system will reject the donor’s marrow.

Casgevy was approved for people ages 12 and up, according to CBS News.

The FDA on Friday also approved the use of Lyfgenia, a second treatment for sickle cell disease that works by genetically modifying a patient’s stem cells.

Lyfgenia is made by Bluebird Bio.

According to NBC News, the new treatments could run an astronomical $2 million per patient according to a report from the Institute for Clinical and Economic Review. The Institute for Clinical and Economic Review is a nonprofit group that helps determine fair prices for drugs the $2 million price doesn’t include the cost of care associated with the treatment.

In people with sickle cell disease, red blood cells, which are usually the shape of a disk, change, becoming more crescent-shaped. The change cells can more easily clump together causing clots and blockages in blood vessels, WebMd explains.

Casgevy stops the process of cells changing into crescent or sickle shapes by editing the DNA in a patient’s stem cells so that they can no longer change shape.

The process for the treatment is a complicated one that requires blood transfusions and chemotherapy to attack a person’s bone marrow before the altered cells are introduced into the body.