What is Casgevy prescribed for?
Casgevy is prescribed to treat people aged 12 years and older with sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs.
What is the name of the drug and what does it do?
Casgevy pronounced cass-JEH-vee, generic name:(exagamglogene autotemcel) is an innovative treatment specifically designed for adults with Beta-Thalassemia, a genetic blood disorder.
This disorder causes the body to produce less hemoglobin, the protein in red blood cells that carries oxygen throughout the body.
Lower hemoglobin means less oxygen gets to various parts of the body, leading to symptoms like severe fatigue and anemia.
Casgevy uses advanced gene-editing technology to address the genetic cause of this disorder, aiming to increase hemoglobin production and reduce the symptoms of the disease.
How does it work?
Casgevy uses a method called CRISPR/Cas9, often described as “genetic scissors,” to precisely change specific parts of DNA in the patient’s bone marrow cells.
The focus is on editing a gene called BCL11A. By tweaking this gene, Casgevy helps turn on another gene that produces fetal hemoglobin — the type of hemoglobin that babies make in the womb, which is particularly good at carrying oxygen.
For patients with Beta-Thalassemia, increasing fetal hemoglobin can compensate for their genetically flawed adult hemoglobin, reducing symptoms and the need for blood transfusions.
What did the research discover?
The studies on Casgevy found that the therapy significantly increased levels of fetal hemoglobin in patients. This boost in healthy hemoglobin led to a notable decrease in the typical symptoms of Beta-Thalassemia, such as anemia.
Importantly, it also reduced the patients’ reliance on regular blood transfusions, which are commonly used to manage the disease but can lead to complications like iron overload.
This result marks a significant advancement in treating a genetic disease directly at its source.
What are some of the side effects?
- Reactions at the injection site
- Fever and chills
- Fatigue
- Headaches
- Muscle aches
- Nausea
What are the dosage recommendations and how is it prescribed?
Casgevy is given as a one-time infusion. In this process, doctors first collect stem cells from the patient’s blood. These cells are then taken to a lab where they are carefully changed to help them work better.
After these changes are made, the cells are put back into the patient’s body. Before this happens, patients go through a special treatment called “conditioning” that gets their bone marrow ready to accept and grow these new, improved cells.
This whole procedure is quite detailed and needs careful planning and coordination by a team of medical experts. It requires a serious commitment from the patient, but it can provide lasting benefits from just one treatment.
Source:
Highlights of Prescribing Information for CASGEVY. Available from: https://www.fda.gov/media/174615/download Accessed 15 July 2024
