Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease.
[3] The US FDA label contains a black box warning about hematologic malignancy (blood cancer).
[1][3] 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.
[3] Effectiveness was evaluated based on complete resolution of VOEs (VOE-CR) between six and eighteen months after infusion with lovotibeglogene autotemcel.
[3] The FDA granted the application for lovotibeglogene autotemcel priority review, orphan drug, fast track, and regenerative medicine advanced therapy designations.