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FDA approves first gene therapies to treat sickle cell disease

The Centers for Disease Control and Prevention says 100,000 Americans are living with sickle cell disease.
FDA approves first gene therapies to treat sickle cell disease
Posted at 12:23 PM, Dec 08, 2023
and last updated 2023-12-08 13:23:15-05

Two gene therapies that the Food and Drug Administration describes as "milestones" were approved Friday for treatment of sickle cell disease.

The Centers for Disease Control and Prevention says 100,000 Americans are living with sickle cell disease, which disproportionately affects Black and Hispanic individuals.

"The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues," the FDA states. 

The mutation causes red blood cells to change shape, which restricts the flow of blood to vessels, causing severe pain and organ damage. 

The two new therapies edit a person's genes to make sure the body produces blood cells that an adult without the disease would produce, the FDA says. Both therapies have been approved for people 12 years and older. 

 “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," said Dr. Nicole Verdun, director of the FDA's Office of Therapeutic Products.

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People who receive either of the two new treatments will be followed for a long-term study to evaluate the product's safety and effectiveness, the FDA said.

“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health," said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

The FDA notes that in clinical trials, the most common side effects were low levels of platelets and white blood cells, as well as mouth sores.


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