The U.S. Food and Drug Administration is set to approve Exa-cel, a potential cure for sickle cell patients, with an anticipated cost of $2 million.
Exa-cel would be the first approved medicine to use CRISPR gene editing technology. Vertex Pharmaceuticals and CRISPR Therapeutics co-developed the treatment, which could cost around $2 million per patient.
The treatment uses Nobel prize-winning technology called CRISPR to edit a patient’s DNA and to alleviate the symptoms of sickle cell disease. The U.K. approved it under the brand name Casegevy in November.
Approval of Exa-cel by the U.S. Food and Drug Administration would mark a scientific breakthrough about a decade after the discovery of CRISPR and a breakthrough for sickle cell patients desperate for a better option.
The cost of a possible cure
According to Dr. Markus Mapara, director of blood and marrow transplantation at New York Presbyterian/Columbia University Irving Medical Center, it is too soon to call Exa-cel a cure.
However, he showed prospective patients’ charts from clinical trials displaying how many pain crises people experienced before and after the treatment. After treatment, most of the patients had zero pain crises.
Dr. Mapara described the effectiveness of the treatment as “mind blowing”.
Why it matters
Sickle cell disease is an inherited blood disorder where a genetic mutation causes normal full moon shaped red blood cells to form crescent shapes and get stuck inside blood vessels, restricting blood flow and causing excruciating pain.
Nigeria leads the world in the number of sickle cell diseases. 4-6 million Nigerians have sickle cell disease. An estimated 150,000 babies are born every year with sickle cell disease in Nigeria. In the United States, the disease affects about 100,000 people.
Few treatments are available, and the only cure is a bone marrow transplant where a patient receives healthy blood stem cells from a donor. New genetic treatments aim to offer relief while removing the need to find donors.