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Lyfgenia (lovotibeglogene autotemcel) is a prescription medication approved by the U.S. Food and Drug Administration (FDA) to treat people ages 12 and older who have sickle cell disease and a history of vaso-occlusive events.
Lyfgenia is a one-time gene therapy for certain people with sickle cell disease. It is made using your own blood-forming stem cells.
Lyfgenia adds working copies of a modified beta-globin gene to these cells. Beta-globin is part of hemoglobin, the protein in red blood cells that carries oxygen. After treatment, your body can make a type of hemoglobin that helps limit red blood cell sickling.
This may decrease or stop vaso-occlusive events, which happen when sickled red blood cells block blood flow and can cause severe pain.
Doctors prescribe Lyfgenia when a person is 12 years of age or older, has sickle cell disease, and has a history of vaso-occlusive events.
Treatment involves several steps. First, your blood stem cells are collected and sent to a manufacturing site to make Lyfgenia. Before the Lyfgenia infusion, you will receive chemotherapy to prepare your bone marrow.
Lyfgenia is given through an intravenous (IV) infusion into a vein.
Lyfgenia is given one time. The minimum recommended dose is 3 × 106 CD34+ cells per kilogram of body weight. CD34+ cells are blood-forming stem cells.
Your dose is based on your body weight and may come in one to four infusion bags. Each bag is infused over less than 30 minutes.
After the Lyfgenia infusion, you will usually stay at the treatment center for about three to six weeks while your healthcare team closely watches your recovery. 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 Lyfgenia for people ages 12 and older with sickle cell disease and a history of vaso-occlusive events, the most common severe side effects occurred in about 33 percent to 71 percent of people. These include:
Lyfgenia has a boxed warning, the FDA’s strongest safety warning, for blood cancers. A boxed warning does not mean the drug is unsafe for everyone or that everyone faces the same level of risk.
Lyfgenia 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.
Genetix Biotherapeutics, the manufacturer of Lyfgenia, offers the Genetix CARES support program at no cost.
The program offers support from a dedicated Patient Navigator, including help locating Qualified Treatment Centers (QTCs) and understanding insurance coverage and benefits.
To learn more, visit Lyfgenia’s support page, call 833-888-6378, or visit Genetix CARES.
Before treatment with Lyfgenia, your healthcare team will test you for HIV-1 and HIV-2. They will also make sure that a stem cell transplant using your own cells (autologous hematopoietic stem cell transplant) is appropriate for you.
Ask your healthcare team about vaccines. The safety of live viral vaccines during or after Lyfgenia treatment has not been studied. Your vaccination schedule should follow the guidelines used after a stem cell transplant using your own cells and for people with reduced spleen function.
Tell your doctor if you have any allergies to Lyfgenia or any of its ingredients. Allergic reactions can happen during the Lyfgenia infusion. Some ingredients, including dimethyl sulfoxide (DMSO) and dextran 40, may cause serious allergic reactions, including anaphylaxis.
Tell your doctor about all medicines you take, including prescription drugs, over-the-counter medicines, vitamins, and herbal supplements. Some medicines, including hydroxyurea (Droxia), HIV antiretroviral medicines, and iron chelators, may need to be stopped before parts of the Lyfgenia treatment process.
Lyfgenia should not be given during pregnancy. A pregnancy test is required before stem cell mobilization, before chemotherapy used to prepare for treatment, and before Lyfgenia is given.
Lyfgenia is not recommended while breastfeeding. Women who can become pregnant and men who can father a child should use effective birth control from the start of stem cell mobilization through at least six months after Lyfgenia is given.
These answers are fact-checked by our editorial staff.
How effective is Lyfgenia for sickle cell disease?
In a study, 28 of 32 people with sickle cell disease, or 88 percent, had no vaso-occlusive events from month 6 through month 18 after treatment. During the same period, 30 people, or 94 percent, had no severe vaso-occlusive events.
Among all 36 people treated with Lyfgenia in the study, 31 people, or 86 percent, reached the study’s globin response goal. This meant they made enough of the added hemoglobin and had improved total hemoglobin levels for at least six months. Some people had vaso-occlusive events again after the main study period, so results were not the same for everyone.
How long does Lyfgenia take to work for sickle cell disease?
The study did not give one exact time when Lyfgenia starts working. The hemoglobin made after treatment generally increased over time and became stable by about month 6.
The main study measured vaso-occlusive event results from month 6 through month 18 after treatment. The added hemoglobin remained at stable levels through month 24, and a smaller group of 10 people continued to show this hemoglobin through month 48.
Does Lyfgenia interact with other medicines?
No formal drug interaction studies have been done with Lyfgenia. It is not expected to interact with certain liver enzymes or drug transporters. However, some medicines may affect parts of the Lyfgenia treatment process and may need to be stopped before or after treatment.
For example:
Because these timing rules are important, your healthcare team should review your medicines before treatment with Lyfgenia. They will tell you which medicines to stop and when they can be restarted.
What tests or monitoring are needed with Lyfgenia for sickle cell disease?
Lyfgenia requires testing and monitoring before, during, and long after treatment.
Before treatment, your healthcare team will screen you for infections, including HIV-1 and HIV-2. During stem cell collection, platelet counts may also need to be checked.
After Lyfgenia is given, your healthcare team will monitor your blood counts. Neutrophil counts are checked until these infection-fighting white blood cells recover. Platelet counts are checked often until platelets recover.
Because blood cancers have occurred after Lyfgenia treatment, long-term monitoring is important. A complete blood count with differential should be done at least every six months for at least 15 years.
A special test called integration site analysis should also be done at months 6 and 12, and as needed. You should continue to be monitored for life.
On MySickleCellTeam, people share their experiences with sickle cell disease, get advice, and find support from others who understand.
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