Another milestone has been passed in the advancement of specialty pharmacy therapies. The FDA has approved not one, but two, gene therapies for a major disease, Sickle Cell Anemia. Most noteworthy is that one of the approvals offers real proof of concept for CRISPR gene-editing technology.
FIRST….. The FDA has approved a unique therapy, Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics, indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). Casgevy is an autologous genome edited hematopoietic stem cell-based gene therapy.
Sickle cell affects an estimated 100,000 people in the United States. The disease can impact any race, however, the black community is the most affected with 90+% of all diagnosed cases. In sickle cell disease, the body makes flawed, sickle-shaped hemoglobin, impairing the ability of red blood cells to properly carry oxygen to the body’s tissues.
The process for Casgevy therapy requires significant preparation. Stem cells harvested from the patient’s bone marrow are sent to manufacturing facilities where they are edited using CRISPR/Cas9 technology. Once the cells are incubated, they are infused back into the patient during a month-long hospital stay.
Second….. The FDA also approved another therapy, Lyfgenia (lovotibeglogene autotemcel) from bluebird bio. This gene therapy is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene. Lyfgenia is also 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.
The key difference is that Casgevy introduces a manufacturer, gene-edited therapy vs. Lyfgenia which is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene that enables production of adult hemoglobin that does not form into the crescent shape associated with the disease.
Refer to Prescribing Information for each therapy for more detail on these complex processes especially around dosage and administration.
CLICK HERE to access Casgevy label
CLICK HERE to access Lyfgenia label
Neither company disclosed launch pricing at time of approval.
Neither company disclosed distribution plans at this time.
