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Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease.

The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with chemotherapy and underlying disease.

The US Food and Drug Administration (FDA) approved lovotibeglogene autotemcel in December 2023.

Medical uses

Lovotibeglogene autotemcel is indicated for the treatment of people aged twelve years of age and older with sickle cell disease and a history of vaso-occlusive events.

The recipient's blood stem cells are genetically modified to produce HbA (T87Q), a gene-therapy derived hemoglobin A, which is similar to the normal adult hemoglobin produced in persons not affected by sickle cell disease. Red blood cells containing HbA (T87Q) have a lower risk of sickling and occluding blood flow. These modified stem cells are then delivered to the recipient.

The gene therapy is made from the recipient's own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Prior to treatment, the recipient's own stem cells are collected, and then the recipient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in lovotibeglogene autotemcel.

Side effects

The US FDA label contains a black box warning about hematologic malignancy (blood cancer).

History

The safety and effectiveness of lovotibeglogene autotemcel is based on the analysis of data from a single-arm, 24-month multicenter study in participants with sickle cell disease and history of Vaso-occlusive episodes (VOEs) between the ages of twelve and fifty years. Effectiveness was evaluated based on complete resolution of VOEs (VOE-CR) between six and eighteen months after infusion with lovotibeglogene autotemcel. Twenty-eight (88%) of 32 participants achieved VOE-CR during this time period.

The FDA granted the application for lovotibeglogene autotemcel priority review, orphan drug, fast track, and regenerative medicine advanced therapy designations. The FDA granted approval of Lyfgenia to Bluebird Bio Inc.

Society and culture

Economics

The cost effectiveness threshold of the therapy is estimated to be between US$1.35 million to $2.05 million.

Brand names

Lovotibeglogene autotemcel is the international nonproprietary name.

Lovotibeglogene autotemcel is sold under the brand name Lyfgenia.

References

1. ^ "Lyfgenia- lovotibeglogene autotemcel suspension". DailyMed. 22 December 2023. Retrieved 30 December 2023.
2. "Lyfgenia". U.S. Food and Drug Administration (FDA). 8 December 2023. Archived from the original on 8 December 2023. Retrieved 9 December 2023. This article incorporates text from this source, which is in the public domain.
3. ^ "FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease". U.S. Food and Drug Administration (FDA). 8 December 2023. Archived from the original on 8 December 2023. Retrieved 8 December 2023. This article incorporates text from this source, which is in the public domain.
4. Kanter, Julie; Thompson, Alexis A.; Pierciey, Francis J.; Hsieh, Matthew; Uchida, Naoya; Leboulch, Philippe; et al. (January 2023). "Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB -206 study". American Journal of Hematology. 98 (1): 11–22. doi:10.1002/ajh.26741. PMC 10092845. PMID 36161320. S2CID 252544370.
5. Sheridan, Cormac (21 November 2023). "The world's first CRISPR therapy is approved: who will receive it?". Nature Biotechnology. 42 (1): 3–4. doi:10.1038/d41587-023-00016-6. PMID 37989785. S2CID 265350318. Archived from the original on 4 December 2023. Retrieved 4 December 2023.
6. "Bluebird Bio Announces FDA Approval of Lyfgenia (lovotibeglogene autotemcel) for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events" (Press release). Bluebird Bio. 8 December 2023. Retrieved 9 December 2023 – via Business Wire.
7. "ICER Publishes Final Evidence Report on Gene Therapies for Sickle Cell Disease". Institute for Clinical and Economic Review. Archived from the original on 4 December 2023. Retrieved 4 December 2023.
8. World Health Organization (2022). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87". WHO Drug Information. 36 (1). hdl:10665/352794.

Further reading

• Kanter J, Walters MC, Krishnamurti L, Mapara MY, Kwiatkowski JL, Rifkin-Zenenberg S, et al. (February 2022). "Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease". The New England Journal of Medicine. 386 (7): 617–628. doi:10.1056/NEJMoa2117175. PMID 34898139.

External links

• "Lovotibeglogene Autotemcel (Code C176855)". NCI Thesaurus.

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