November 1, 2023 – A groundbreaking gene therapy that may potentially cure sickle cell anemia is heading for FDA approval in December.
The treatment, called Exa-Cel, received positive safety reviews from a key FDA advisory panel inside seven hours meet on Tuesday. Exa-cel is a gene editing treatment that alters the body's process for making red blood cells.
Sickle cell anemia causes abnormal blood flow to the body, which may result in eye problems, infections and severe pain. The disease is extremely related to early death, often from stroke or infection. People with this disease have a median life expectancy of 43 years CDC.
Currently, the one cure is a bone marrow transplant, which is commonly not an option as a result of the rarity of finding an acceptable donor.
The latest treatment, made by CRISPR Therapeutics and Vertex Pharmaceuticals, involves a one-time infusion-like process using an individual's own stem cells. Before the infusion, the person's stem cells are processed in a laboratory and the person must undergo chemotherapy, in response to a summary of the Sickle Cell Anemia Association of America.
If approved, the treatment will likely be available to people ages 12 and older. Sickle cell anemia is especially devastating for young children and is one in all the leading causes of death in children under 5 worldwide, in response to an evaluation published In The Lancet Hematology last summer.
The disease affects greater than 100,000 people within the United States and is most typical in people of black or African descent. According to the WHO, about one in 365 babies of African descent is born with sickle cell anemia National Institutes of Health.
The latest treatment is predicted to cost thousands and thousands of dollars. The New York Times and the Sickle Cell Disease Association of America each reported. About 20,000 people within the U.S. with severe sickle cell disease could be eligible, CRISPR Therapeutics said in a news release.