Two New Treatments for Sickle Cell Disease Approved by the FDA
On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved the first CRISPR-based medicine to treat sickle cell disease. The treatment, called Casgevy (exagamglogene autotemcel), is a cell-based gene therapy. It's the first FDA-approved therapy that uses CRISPR/Cas9, a type of genome editing technology. CRISPR/Cas9 can be directed to cut DNA in targeted areas, enabling the ability to accurately edit (remove, add, or replace) DNA where it was cut. The modified blood stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery. In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.I reached out to the Sickle Cell Foundation of Georgia and their executive director Tabatha McGee to learn about these new developments.
I’ve been excited about the prospects of CRISPR for a while and find it interesting that the first disease to use this opportunity is Sickle Cell. I asked McGee about the cost of the treatment and if it will be accessible to the general population. She explains that currently the Casgevy treatment is only available to patients who meet the guidelines. This means that they experience chronic disability. McGee goes on to explain that the Casgevy treatment costs $3.1 Million.
Both treatments use the patient’s blood cells which are modified and returned to the body. I asked about the second Lyfgeniaand McGee shares that this treatment costs $2.1 Million. While the costs are high they are in communication with the Center for Medicare and Medicaid services about strategies to bring coverage through those insurance offerings.
Even though the cost means less accessibility, McGee explains that these two new treatments are options that include healthy lifestyles, bone marrow transplants and provides more options for treatment. These are the first new approved treatments in several decades.
McGee also reminds us that a new Wellness Center will open at their headquarters at 2391 Benjamin E. Mays Drive early in 2024.
For more information on the Georgia Sickle Cell Foundation
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Listen to the conversation about the new treatments FDA approval of gene therapies for sickle cell disease: Q&A with NHLBI Director Dr. Gary Gibbons and NHLBI’s Division of Blood Diseases and Resources Director Dr. Julie Panepinto