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Casgevy (exagamglogene autotemcel) is a prescription medication approved by the U.S. Food and Drug Administration (FDA) for people 2 years of age and older who have sickle cell disease with repeated episodes called vaso-occlusive crises (VOCs).
Casgevy is a one-time gene therapy for people two years of age and older who have sickle cell disease with repeated vaso-occlusive crises. VOCs are painful episodes caused when sickled red blood cells block blood flow.
Casgevy is made using your own blood-forming stem cells. These cells are changed using gene-editing technology. The change lowers the activity of BCL11A, which helps your body make more fetal hemoglobin. Fetal hemoglobin is a type of hemoglobin that can help keep red blood cells from sickling. This can eliminate VOCs in people with sickle cell disease.
Treatment with Casgevy involves several steps. First, your blood-forming stem cells are collected. The cells are then sent to a manufacturing site, where they are used to make Casgevy.
Before you receive Casgevy, you will be given chemotherapy. This treatment makes room in your bone marrow for the edited stem cells.
Casgevy is given once through a vein. Your dose may come in one or more vials. Each vial is given through a central venous catheter and must be administered within 20 minutes after it is thawed.
The minimum recommended dose of Casgevy is 3 million CD34-positive cells per kilogram of body weight. CD34-positive cells are blood-forming stem cells. Your dose is based on your body weight.
After the Casgevy infusion, you will stay in the hospital while your healthcare team watches your recovery closely. This can take four to six weeks, but the time can vary. Your healthcare provider will decide when it is safe for you to go home.
This information is based on prescribing information, but your healthcare provider may tailor your treatment plan. Always follow their guidance.
In clinical studies of Casgevy, the most common side effects were reported in about 27 percent to 86 percent of people. These include:
Casgevy can cause serious side effects that may require immediate medical attention. These include:
Get medical help right away if you think you are having a serious reaction.
Vertex Pharmaceuticals Incorporated, the manufacturer of Casgevy, offers the Vertex Connects Patient Support Program. The program helps you understand insurance coverage and navigate the treatment process.
Vertex Connects also offers support throughout your treatment journey, including access to a care manager, educational resources, treatment coordination, and eligibility screening for additional support programs such as travel and lodging assistance and fertility support for eligible people with sickle cell disease and Medicaid coverage in certain states.
To learn more, visit the Vertex Connects website or call 833-837-8395.
Before treatment with Casgevy, your healthcare team will check whether a blood-forming stem cell transplant using your own cells is right for you.
You will also be tested for human immunodeficiency virus (HIV)-1, HIV-2, hepatitis B, hepatitis C, and other infections based on local guidelines. Casgevy should not be used in people with active HIV-1, HIV-2, hepatitis B, or hepatitis C.
If you can become pregnant, you must have a negative pregnancy test before each stem cell mobilization cycle and again before the chemotherapy used to prepare your bone marrow.
The safety of live viral vaccines during or after Casgevy treatment has not been studied.
Casgevy contains dimethyl sulfoxide, also called DMSO, and dextran 40. These ingredients can cause allergic reactions, including serious reactions. Your healthcare team will watch for these reactions during and after the infusion.
Tell your doctor about all medicines you take, including prescription drugs, over-the-counter medicines, vitamins, and herbal supplements.
Some medicines need to be stopped before treatment. Hydroxyurea must be stopped at least eight weeks before each stem cell mobilization cycle and conditioning. Iron chelators must be stopped at least seven days before conditioning. Follow your healthcare provider’s instructions about your medicines.
Casgevy is also FDA-approved to treat people two years of age and older with transfusion-dependent beta thalassemia.
Casgevy must not be given during pregnancy because of the risks from the chemotherapy used before treatment. People who can become pregnant and people who can father a child should use effective birth control from the start of stem cell mobilization through at least six months after Casgevy is given.
Breastfeeding should be stopped during conditioning. After the Casgevy infusion, talk with your doctor about whether to breastfeed.
These answers are fact-checked by our editorial staff.
How effective is Casgevy for sickle cell disease?
In one study of people age 12 years and older, 29 of 31 evaluable people, or 93.5 percent, had no severe VOCs for at least 12 straight months within the first 24 months after Casgevy infusion.
In another study, all eight evaluable children age 5 years to younger than 12 years had no severe VOCs for at least 12 straight months.
Among people evaluated for hospitalizations, all 30 people age 12 years and older and all eight children in the younger group had no hospitalizations due to severe VOCs for at least 12 straight months.
There were no cases of graft failure or graft rejection in these studies.
How long does Casgevy take to work for sickle cell disease?
The study does not give an exact time when Casgevy starts working.
In the studies, increases in total hemoglobin and fetal hemoglobin were seen by month 3 after Casgevy infusion. The main study outcomes for severe VOCs began to be measured 60 days after the last red blood cell transfusion given for support or sickle cell disease management.
In the study of people age 12 years and older, those who met the main treatment goal had a median of 22.2 months without a severe VOCs.
Does Casgevy interact with other medicines?
No formal drug interaction studies have been done with Casgevy. Casgevy is not expected to interact with certain liver enzymes or drug transporters.
Some medicines need to be stopped during parts of the Casgevy treatment process:
Your healthcare team will review your medicines and tell you which ones to stop and when.
What tests or monitoring are needed with Casgevy for sickle cell disease?
Before your cells are collected, your healthcare team will screen you for HIV-1, HIV-2, hepatitis B, hepatitis C, and other infections based on local guidelines.
If busulfan is used to prepare your bone marrow for treatment, blood samples are taken to measure busulfan levels. Your healthcare team may adjust the dose based on these results.
After Casgevy infusion, your healthcare team will monitor your neutrophils, which are a type of white blood cell, and your platelets, which help your blood clot. Platelet levels are checked often until they recover. Your healthcare team may also order blood counts and other tests if you have signs of bleeding.
On MySickleCellTeam, people share their experiences with sickle cell disease, get advice, and find support from others who understand.
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