Lyfgenia FDA Approval History

Last updated by Judith Stewart, BPharm on Dec 19, 2023.

FDA Approved: Yes (First approved December 8, 2023)
Brand name: Lyfgenia
Generic name: lovotibeglogene autotemcel
Dosage form: Suspension for Intravenous Infusion
Company: Bluebird Bio, Inc.
Treatment for: Sickle Cell Disease

Lyfgenia (lovotibeglogene autotemcel) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events.

• Sickle cell disease (SCD) is a complex and progressive genetic disease where high concentrations of sickle hemoglobin in red blood cells cause red blood cells to become sickled, sticky, and rigid with a shorter life span, which manifests acutely as hemolytic anemia, vasculopathy, and vaso-occlusion. SCD is characterized by debilitating and unpredictable pain crises, irreversible damage to vital organs, and early death.
• Lyfgenia is a one-time gene therapy designed to add functional copies of a modified form of the β-globin gene (β^A-T87Q-globin gene) into a patient’s own hematopoietic stem cells. Once patients have the β^A-T87Q-globin gene, their red blood cells can produce anti-sickling hemoglobin (HbA^T87Q) that decreases the proportion of sickle hemoglobin (HbS), with the goal of reducing sickled red blood cells, hemolysis, and other complications.
• Lyfgenia is infused back into the patient as part of an autologous hematopoietic stem cell transplant (HSCT) after myeloablative busulfan conditioning.
• Warnings and precautions include delayed platelet engraftment, neutrophil engraftment failure, insertional oncogenesis, and hypersensitivity reactions.
• Adverse reactions include stomatitis, thrombocytopenia, neutropenia, febrile neutropenia, anemia, and leukopenia.

Development timeline for Lyfgenia

Further information

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