Nov. 1, 2023 – A breakthrough genetic therapy that can potentially cure sickle cell disease is poised for approval by the FDA in December.
The treatment, called exa-cel, received a positive critique of its safety from an important FDA advisory panel during a 7-hour meeting on Tuesday. Exa-cel is a gene-editing treatment that changes the body’s process for making red blood cells.
Sickle cell disease leads to abnormal blood flow in the body that can cause eye problems, infections, and severe pain episodes. The disease is highly linked to early death, often from a stroke or infection. People with the disease have an average life span of 43 years, according to the CDC.
Currently, the only cure is a bone marrow transplant, which is often not an option due to the rarity of finding a matched donor.
Made by CRISPR Therapeutics and Vertex Pharmaceuticals, the new treatment involves a one-time infusion-like process using a person’s own stem cells. Before the infusion, the person’s stem cells are edited in a lab and the person must have chemotherapy, according to a summary from the Sickle Cell Disease Association of America.
If approved, the treatment will be available for people age 12 and older. Sickle cell disease is particularly devastating for young children and is among the top causes of death among children under age 5 worldwide, according to an analysis published in The Lancet Haematology this past summer.
The disease affects more than 100,000 people in the U.S., and it is most common in people who are Black or of African ancestry. About 1 in 365 babies who are of African descent are born with sickle cell disease, according to the National Institutes of Health.
The new treatment is expected to cost millions of dollars, The New York Times and Sickle Cell Disease Association of America both reported. About 20,000 people in the U.S. with severe sickle cell disease would be eligible, CRISPR Therapeutics said in a news release.