FDA approves first gene-editing treatment for sickle cell disease

Sickle cell gene editing treatment overview: 

• Who: The U.S. Food and Drug Administration (FDA) has approved the gene-editing treatment, Casgevy, which was co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics. 
• Why: Casgevy, the first gene-editing treatment marketed in the US, will be used to treat individuals with sickle-cell disease. 
• Where: An estimated 100,000 Americans around the US are affected by sickle cell disease.

The U.S. Food and Drug Administration (FDA) has approved Casgevy, the first gene-editing treatment marketed in the country, with the therapy set to be used to treat sickle cell disease.  

The FDA has approved the gene editing treatment for individuals age 12 and older, the month after regulators in the UK did the same, CNBC reports. 

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

Vertex Pharmaceuticals and CRISPR Therapeutics reportedly co-developed Casgevy, which will cost $2.2 million to administer per patient and is now approved around a decade after CRISPR discovered technology allowing it to edit human DNA. 

An estimated 100,000 Americans are reportedly affected by sickle cell disease, an inherited disease that causes red blood cells to become misshapen, get stuck inside blood vessels and ultimately restrict blood flow, causing pain crises. 

Casgevy able to eliminate pain crises in majority of patients who were part of clinical trials 

Casgevy works by editing an individual’s DNA to have it turn on fetal hemoglobin — a protein that normally turns off shortly after a person is born — to help prevent red blood cells from becoming misshapen, according to CNBC. 

In clinical trials, Casgevy was reportedly able to eliminate pain crises in most of the patients who underwent the gene-editing treatment.

While Casgevy is administered only once, the process takes months to complete, with extracted blood cells sent to Vertex’s lab to be genetically modified before being infused into a patient who has undergone a few days of chemotherapy to clear out old cells, and who must then spend weeks in the hospital recovering, CNBC reports. 

Vertex reportedly said it plans to take the lead on launching Casgevy, with the biotech company estimating around 16,000 people with a severe case of sickle cell disease will be eligible to receive the gene-editing treatment. 

The FDA proposed rescinding a regulation authorizing the use of brominated vegetable oil as a food additive last month, arguing the results of recent toxicology studies provided the agency with “conclusive scientific evidence” to support the removal. 

Do you believe you would qualify for the Casgevy gene-editing treatment Let us know in the comments.

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