FDA approves new sickle cell disease treatment. What does it mean for Georgia?

ATLANTA — The U.S. Food and Drug Administration announced Friday that they’d approved a new gene-editing therapy to treat Americans with sickle cell disease.

About 100,000 Americans have sickle cell disease, according to the U.S. Centers for Disease Control and Prevention.

The CDC estimated that about one in every 365 African-American children are born with SCD, while the same is true for one in every 16,300 Hispanic-American births.

Georgia is one of 11 states in the U.S. where the CDC is tracking SCD data. Their tracker shows the counties across Georgia with a dense population of SCD patients, saying nearly half of those individuals live in five metro Atlanta counties.

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Sickle cell disease is an inherited red blood cell disorder, according to the CDC, where the cells are C-shaped instead of round due to abnormal hemoglobin, the part of the blood cell that transports oxygen.

Patients with SCD have blood cells that die too fast, causing a constant shortage of red blood cells, in addition to their shape contributing to blockages in blood vessels, which can cause pain and slow blood flow, the CDC said.

As an inherited disorder, meaning it is genetic, sickle cell is not infectious to others, but instead is passed on from parent to child. The treatment approved for human use by the FDA on Friday would essentially edit genetic code to remove the mutation that causes sickle cell disease.

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Casgevy and Lyfgenia, the gene therapies now approved for use on human patients, will be available to patients as young as 12-years-old and uses CRISPR, (Clustered Regularly Interspaced Short Palindromic Repeats) to edit genomes through what’s known as Cas9 technology, according to the FDA.

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

For thousands of Georgia patients, the approval opens up a new option to treat the disease and improve health and living conditions impacted by having sickle cell disease.

In the Atlanta metro area, Clayton, Cobb, DeKalb, Fulton and Gwinnett counties accounted for 45% of all sickle cell patients in the state, though the numbers provided by CDC are from 2018, the most recent numbers available. The same data showed more than 90% of Georgia’s counties have patients with SCD.

Following the FDA approval, President Joe Biden praised the decision, saying in part that “this significant medical advancement holds tremendous promise for developing additional life-saving treatments and it gives hope to the millions of Americans who live with other rare diseases.”

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