The US Food and Drug Administration (FDA) has approved the first gene editing therapy ever to be used in humans, for sickle cell disease.
The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, received FDA’s approval on Friday. It was approved for patients who are 12 years and above. It works by editing the DNA found in a patient’s stem cells to remove the gene that causes the disease.
Vertex Pharmaceuticals and its partner CRISPR Therapeutics said on Friday their sickle cell disease gene therapy Casgevy would be available at a list price of $2.2 million in the United States. This price doesn’t include the cost of care associated with the treatment, such as stay in the hospital or chemotherapy.
The agency also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drug maker Bluebird Bio. Both treatments work by genetically modifying a patient’s own stem cells.
On its part, Bluebird bio said it has set a list price of $3.1 million for its treatment, Lyfgenia.
Bone marrow transplant from a donor has been the only known cure for sickle cell disease until now. It however associated by the risk of rejection by the recipient’s immune system.
Although Casgevy is technically a one-time treatment, there are multiple phases to the journey. It requires a number of steps that span months before the patient actually gets the modified stem cells.
The treatment process begins with a series of blood transfusions over three to four months, after which the stem cells are extracted from the patient’s bone marrow and sent off to a lab where they are edited, Hanna said.
Then chemotherapy is used to destroy the patient’s bone marrow to ensure that no flawed stem cells remain in the body before the stem cells can be reinfused into the patient.
This is followed by another month or two in the hospital to allow the cells to grow and the patient to recover.
The clinical trial of the treatment included 46 people in the U.S. and abroad, 30 of whom had at least 18 months of follow-up care after the treatment. Of those, the treatment has been successful in 29.
The US has an estimated 100,000 individuals living with sickle cell disease. It causes pain and can lead to premature death.
According to reports, Nigeria has the largest population of persons affected with sickle cell disease globally. 90% of the world’s sickle cell disease population lives in Nigeria, India, and the Democratic Republic of Congo.
Ifunanya Ikueze is an Engineer, Safety Professional, Writer, Investor, Entrepreneur and Educator.