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Lyfgenia

Pronunciation: lif-JEN-ee-uh
Generic name: lovotibeglogene autotemcel
Dosage form: suspension for intravenous infusion
Drug class: Miscellaneous uncategorized agents

Medically reviewed by Carmen Pope, BPharm. Last updated on Jan 4, 2024.

Lyfgenia (lovotibeglogene autotemcel) is a gene therapy that is given as a one-time intravenous suspension to treat adults and children aged 12 years and older who have sickle cell disease (SCD) with a history of vaso-occlusive crises (VOCs). VOCs are when sickled red blood cells block blood flow, depriving tissues of oxygen. Lyfgenia uses a common, well-studied viral vector to deliver genetic modifications to a patient’s blood stem cells, so no donor is needed.

SCD is a genetic, inherited, lifelong disease caused by an alteration in one of the genes in the red blood cell, the beta-globin gene, that causes the normal disc-shaped red cells to take the shape of a sickle, causing anemia and VOCs, like a pain crisis.

Lyfgenia uses a replication-incompetent, self-inactivating, lentiviral vector (LVV) to add functional copies of the beta-globin gene to the patient’s own blood stem cells, which results in the production of anti-sickling hemoglobin that may decrease or stop vaso-occlusive events.

Lyfgenia was FDA approved on December 8, 2023.

Warnings

Lyfgenia may cause hypersensitivity reactions during the infusion. Your doctor will monitor you for these.

There is a risk of neutrophil engraftment failure after HSC transplant and Lyfgenia administration which may require rescue treatment with your collection of unmodified CD34+ cells.

Longer median platelet engraftment times were observed with Lyfgenia treatment compared to an allogeneic HSC transplant. Until then there is an increased risk of bleeding. Your doctor will monitor you for bleeding and check your platelet counts. In clinical trials, 2 patients required more than 100 days to achieve platelet engraftment.

There is a potential risk for blood cancer because Lyfgenia uses a viral vector. Because of this risk, your doctor will monitor you every 6 months for a minimum of 15 years.

Before taking

Chemotherapy received before the administration of Lyfgenia may reduce your chances of becoming pregnant or fathering a child. You should consider discussing options for fertility preservation with your doctor before treatment with Lyfgenia.

Pregnancy/Lactation

A negative serum pregnancy test must be confirmed before the start of mobilization and re-confirmed before conditioning procedures and before Lyfgenia administration.

Do not administer Lyfgenia to women who are pregnant, planning a pregnancy, or breastfeeding. Contact your healthcare provider right away if you inadvertently become pregnant.

Women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra-uterine device or combination of hormonal and barrier contraception) from the start of mobilization through to at least 6 months after administration of Lyfgenia.

Lyfgenia pregnancy and breastfeeding warnings (more detail)

How is Lyfgenia administered?

Lyfgenia is made specifically for each patient. You will be admitted to a treatment center during this process.

Before treatment, your healthcare providers will give you other medicines, including a chemotherapy medicine (given in the vein), as part of your treatment. Talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment.

There are 4 main steps involved with Lyfgenia treatment.

Step 1: Lyfgenia is made specifically for you from your blood stem cells. Your healthcare provider will collect your blood stem cells through a procedure/process called mobilization and apheresis. This process takes approximately one week and may need to be repeated to obtain a sufficient number of cells. Back-up stem cells (or rescue cells) are also collected and stored at the treatment center. This is a precaution in case there is a problem in the treatment process. If this happens, your backup stem cells will be given back to you. If you receive back-up cells, you will have no benefit from Lyfgenia.

Step 2: Your blood stem cells will be sent to a manufacturing site where they are used to make your Lyfgenia. It typically takes 10 to 15 weeks from the time your cells are collected to make and test the infusion before it is shipped to your healthcare provider, but the time may vary.

Step 3: Before you receive Lyfgenia, your healthcare provider will give you chemotherapy for a few days to make room in the bone marrow. You will be admitted to the treatment center for this step and remain there until after the infusion.

Step 4: Lyfgenia is given by an intravenous infusion (into your vein). You may receive more than one bag and each bag is infused in 30 minutes or less. After the infusion, you will stay in the treatment center for approximately 3-6 weeks so that your healthcare team can closely monitor your recovery.

Detailed Lyfgenia dosage information

What should I avoid after receiving Lyfgenia?

Do not donate blood, organs, tissues, or cells.

What are the side effects of Lyfgenia?

Treatment with Lyfgenia may increase your risk of developing blood cancer. These cancers may develop many years after treatment with Lyfgenia and can be life-threatening and/or cause death. Talk to your doctor about the benefits and risks of Lyfgenia and your treatment options. Because of the risk of cancer, you will need to be monitored at least every 6 months for a minimum of 15 years after your Lyfgenia infusion. Monitoring will include blood tests and additional testing if necessary. Blood cancer may cause no symptoms, or symptoms can be general. Call your healthcare provider right away if you develop any of these signs or symptoms:

If you are diagnosed with cancer, have your treating physician contact Bluebird Bio at 1-833-999-6378.

You may also experience side effects associated with other medicines administered as part of the Lyfgenia treatment regimen. Talk to your physician regarding those possible side effects.

Possible side effects of Lyfgenia may include low blood pressure or hot flushes on the day of treatment.

Following treatment, possible side effects include:

These are not all the possible side effects of Lyfgenia. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or to Bluebird Bio at 1-833-999-6378.

Lyfgenia side effects (more detail)

Following Lyfgenia treatment

Keep your regular check-ups with your healthcare provider, including blood tests at least every 6 months, to detect any adverse effects and to confirm that Lyfgenia is still working.

Talk to your physician about enrolling in a post-marketing study to assess the long-term safety of Lyfgenia including the risk of blood cancers occurring after treatment.

Lyfgenia will not give you a human immunodeficiency virus (HIV) infection although it may cause a  false-positive HIV test result by some commercial tests (specifically, a PCR-based test). If you need to have an HIV test, talk with your healthcare provider about the appropriate test to use.

Talk to your healthcare provider about any concerns. You can ask your healthcare provider for information about Lyfgenia that is written for healthcare professionals. For more information, go to Lyfgenia.com or call 1-833-666-2583 for Bluebird Patient Services (my Bluebird support).

What other drugs will affect Lyfgenia?

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, antacids, and herbal supplements.

Your doctor will advise you to avoid the following medications:

Lyfgenia will not give you a human immunodeficiency virus (HIV) infection although it may cause a false-positive HIV test result by some commercial tests (specifically, a PCR-based test). If you need to have an HIV test, talk with your healthcare provider about the appropriate test to use.

This is not a full list of interactions. See the prescribing information for a full list.

Lyfgenia drug interactions (more detail)

Storage

Keep the infusion bag(s) in the metal cassette(s) and store in the vapor phase of liquid nitrogen at less than or equal to -140°C (≤ -220°F) until ready to thaw and administer.

Thaw Lyfgenia before the infusion. Do not re-freeze after thawing. Do not irradiate Lyfgenia, because this could lead to inactivation.

Ingredients

Supplied as one to four 20 mL infusion bags. The infusion bags contain a frozen suspension of genetically modified autologous cells, enriched for CD34+ cells. 

Each infusion bag is individually packed within an overwrap in a metal cassette. It is shipped from the manufacturing facility to the treatment center storage facility in a cryo shipper, which may contain multiple metal cassettes intended for a single patient. A Lot Information Sheet is affixed inside the shipper.

Match the identity of the patient with the patient identifiers on the metal cassette(s), infusion bag(s), and Lot Information Sheet upon receipt.

Manufacturer

Bluebird Bio, Inc.

References

  1. Product Label

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer