The Food and Drug Administration (FDA) on Friday approved a new therapy for treating sickle cell disease, with this move also marking the first instance of CRISPR gene editing receiving approval from federal regulators.
The FDA approved two new treatments for sickle cell disease (SCD) on Friday, Casgevy and Lyfgenia.
Casgevy, also known as exa-cel, is developed through a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics. The treatment involves taking a sickle cell patient’s own stem cells, editing them to create more fetal hemoglobin and transplanting them back into the individual.
When more fetal hemoglobin is produced, red blood cells don’t become “sickle” shaped, which is what causes the complications and pain associated with SCD. About 100,000 people in the U.S. have SCD, with the disease mostly affecting Black patients.
The FDA has approved the treatment for SCD patients 12 years old and up.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” Nicole Verdun, director of the FDA’s Office of Therapeutic Products, said in a statement.
A bone marrow transplant has long been the only curative treatment for SCD, with an ideal donor usually being a fully related sibling. There is, however, only a 1-in-4 chance that a sibling will be a match and most patients don’t have this option. Casgevy essentially makes a patient their own donor.
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