- The FDA approved the first gene therapies for sickle cell anemia.
- The two approved treatments, Casgevy and Lyfgenia, will cost $2.2 million and $3.1 million.
- Casgevy is the first therapy to use the CRISPR gene editing tool that won the Nobel Prize.
The FDA approved the first gene therapy treatments for sickle cell disease on Friday, but it will cost patients millions of dollars.
One of the approved therapies, developed by Vertex Pharmaceuticals and called Casgevy, is the first of its kind to use the CRISPR gene-editing tool, according to the FDA.
Emmanuelle Charpentier and Jennifer A. Doudna won the Nobel Prize in chemistry in 2020 for their work creating the CRISPR tool.
Sickle cell is an inherited blood disorder. It affects the shape of the red blood cells that carry oxygen through the body, according to the Mayo Clinic. The malformed blood cells caused by the disease can become rigid, slowing and blocking blood flow.
The CRISPR technology works by cutting DNA in targeted areas and removing, adding, or replacing DNA where it was cut, the FDA says. In the Casgevy therapy, modified blood stem cells are transplanted back into the patient, where they multiply within the bone marrow and increase the production of fetal hemoglobin, which prevents the sickling of red blood cells, according to the agency.
Vertex and CRISPR Therapeutics said in a statement that about 16,000 sickle cell patients who are over the age of 12 may be eligible for the therapy, which offers a “potential of a functional cure for their disease.”
That therapy, however, could cost a single patient more than $2.2 million, not including the cost of associated care, such as a hospital stay or chemotherapy, according to an SEC filing.
Rabi Hanna, a pediatric hematologist-oncologist at the Cleveland Clinic who previously served on the advisory board for Vertex, told NBC that the therapy could be “an equalizer” for people with sickle cell. Many people suffering from the disease are unable to work.
“We really have to make sure that it is accessible,” he told NBC.
The second treatment the FDA approved on Friday is called Lyfgenia. Lyfgenia modifies a patient’s blood stem cells and transplants them, but it instead adds normal hemoglobin that is uninfected with the disease to the cells so that they have a lower risk of sickling, according to the FDA.
Lyfgenia will come with an even higher price tag of $3.1 million, bluebird bio, the biotech company that developed the treatment, said in a news release.
