Up until now, the only real treatment has been a stem cell or bone marrow transplant. The new exa-cel treatment under FDA consideration can use the patient's own stem cells.
If approved, exa-cel would be the first FDA-approved treatment that uses genetic modification called CRISPR.sickle cell
The independent committee is helping the FDA think through how it should evaluate a treatment called exa-cel that could potentially cure people of sickle cell disease, a painful and deadly disease with no universally successful treatment. This was an ongoing discussion. There was no vote or decision about the therapy, but the discussion likely moves the US one step closer to approving a groundbreaking new treatment that uses gene editing.
Up until now, the only real treatment has been a stem cell or bone marrow transplant. For stem cells, fewer than 20% of patients have an appropriately matched donor, the FDA said, and the transplants are risky and may not work. Sometimes a transplant can kill the patient.The new exa-cel treatment under FDA consideration can use the patient's own stem cells.
An FDA presentation to the panel suggested the agency may have some questions about the data. It called a lack of confirmatory testing "concerning." It also noted the study's small patient size.
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