FDA Approves Casgevy™ – First-ever approval of a CRISPR-based gene-editing therapy in the U.S. for the Treatment of Sickle Cell Disease
Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that the U.S. Food and Drug Administration has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). This approval means that for the first time, approximately 16,000 patients with SCD may be eligible for a durable one-time therapy that offers the potential of a functional cure for their disease by eliminating severe VOCs and hospitalizations caused by severe VOCs.
Its approval in the U.S. and U.K. caps a decade of remarkable scientific progress translating CRISPR from academic breakthrough to new medicine.
CASGEVY is a genome-edited cellular therapy consisting of autologous CD34+ hematopoietic stem cells (HSCs) edited by CRISPR/Cas9 technology at the erythroid specific enhancer region of the BCL11A gene. It is intended for one-time administration for people aged 12 years and older with (SCD). The hematopoietic stem cell transplant procedure uses the patient’s own CD34+ cells which are modified to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin (HbF) production. HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with SCD.
Sickle cell disease (SCD) is a debilitating, progressive and life-shortening disease. SCD patients report health-related quality of life scores well below the general population, and the lifetime health care costs in the U.S. of managing SCD for patients with recurrent VOCs is estimated between $4 and $6 million. SCD is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. Due to misshapen or “sickled” red blood cells, SCD causes severe pain, organ damage and shortened life span. SCD requires a lifetime of treatment and results in a reduced life expectancy. In the U.S., the median age of death for patients living with SCD is approximately 45 years.
A cure for SCD today is a stem cell transplant from a matched donor, but this option is only available to a small fraction of patients living with SCD because of the lack of available donors.
