Almost a year once approved, patients are already receiving their first therapy using the Nobel Prize-winning Crispr technology.
The gene-modifying treatment, called Casgevy, is for people with sickle cell disease and a related blood disorder called beta thalassemia. British regulators approved the treatment in November 2023, followed in December in the US and Europe. Vertex, the pharmaceutical company that markets Casgevy, announced in a call for proposals on November 5 that the first person to receive Casgevy outside a clinical trial received a dose in the third quarter of this year. The company recorded revenue of $2 million from this patient. (Casgevy debuted in the US with a price tag of $2.2 million.)
“Cagevy has been enthusiastically received by patients, physicians and policymakers, and the rollout is gaining momentum in all regions,” Stuart Arbuckle, Vertex’s chief operating officer, said on the call. Additional patients have access to treatment commercially, he added.
When WIRED contacted Vertex via email, spokeswoman Eleanor Celeste declined to provide the exact number of patients who received Casgevy. However, the company said 40 patients had cells collected while awaiting treatment, up from 20 patients last quarter.
With sickle cell disease and beta-thalassemia, patients do not produce fit hemoglobin, a substance found in red blood cells that is responsible for carrying oxygen around the body. Errors in the hemoglobin gene are to blame. As a result, people with sickle cell disease have tough, crescent-shaped red blood cells that stick together and block blood flow, causing extreme bouts of pain. These pain crises can last for hours or days and may send the patient to the hospital. In beta-thalassemia, the body does not produce enough hemoglobin, which leads to anemia. People with severe beta-thalassemia require regular blood transfusions every few weeks throughout their lives.
Casgevy uses Crispr to modify the patient’s own cells so that they produce a fit type of hemoglobin.
A delay in patients taking Casgevy is not necessarily unexpected as the treatment is sophisticated to administer and only some hospitals are able to carry out the procedure. On an earnings call last week, Arbuckle said 45 treatment centers are currently authorized to administer Casgevy, and Vertex expects that number to grow to about 75 worldwide.