Genetic Therapy Approved for Patients With Sickle Cell Disease

The US Food and Drug Administration (FDA) has approved lovotibeglogene autotemcel (lovo-cel), a cell-based gene therapy for patients aged 12 years and older with sickle cell disease (SCD) with a history of vaso-occlusive events. Lovo-cel was developed by bluebird bio, Inc., under the trade name Lyfgenia.

“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, MD, PhD, Director of the FDA Center for Biologics Evaluation and Research.

Lovo-cel is produced from a patient’s own blood cells, which are modified and transplanted back into the patient to modulate the hemoglobin mutation that causes the sickling of red blood cells in SCD. Lovo-cel specifically uses a lentiviral vector to implant a gene sequence that allows patients with SCD to produce hemoglobin A^T87Q, a type of hemoglobin derived from gene therapy that is comparable to the normal hemoglobin A in adults without SCD.

The primary data supporting the approval of lovo-cel came from a single-arm, multicenter, 24-month trial that enrolled 32 patients aged from 12 to 50 years old with SCD and a history of vaso-occlusive events. Overall, 28 (88%) participants achieved total resolution of vaso-occlusive events in six to 18 months after lovo-cel infusion.

The most common side effects included stomatitis, febrile neutropenia, and low levels of platelets and white and red blood cells—a side effect profile consistent with chemotherapy and underlying disease.

“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health,” stated Dr. Marks.