FDA approves groundbreaking CRISPR-based gene therapies for sickle cell disease
CHICAGO - The Food and Drug Administration announced its approval of the first genetic therapy treatment for sickle cell disease, utilizing the gene-editing tool CRISPR.
The approval, disclosed on Friday morning, includes two milestone treatments named Casgevy and Lyfgenia. These therapies represent the first cell-based gene therapies for sickle cell disease treatment in patients aged 12 years and older.
Sickle cell disease, affecting around 100,000 people in the U.S., is prevalent among African Americans and constitutes a group of inherited blood disorders. Jalen Matthews, a sickle cell patient, shared her challenging journey, including chronic pain, frequent hospital treatments, a blood clot, and experiencing a stroke at the age of twelve.
"I had to learn how to walk again, feed myself, clothe myself, basically learn how to do everything all over again," said Matthews.
The disease results from a mutation leading to abnormal hemoglobin. The newly approved gene-editing treatment targets and corrects this mutation.
"The clinical trials done were done with sickle cell patients showed an amazing effect," said Dr. Nicole Verdun, an FDA Super Office Director. "They had a reduction in pain and episodes that lead to severe and debilitating disease."
Dr. Nicole Verdun, a Chicagoan and FDA Director of the Office of Therapeutic Products, played a crucial role in obtaining FDA approval for the two gene-editing treatments. These treatments can be performed in a single doctor's visit for patients aged 12 and older.
During the two-year follow-up on clinical trial patients, the majority being African American due to the group's disproportionate impact, doctors observed promising results. Although the therapies show great potential, medical professionals acknowledge that there are still uncertainties.
"What this has been able to show is that in the data that we have is that the people that have had this treatment for pain have gone for practically 0 hospitalizations for pain," said Dr. Lewis Hsu, Chief Medical Officer for the Sickle Cell Disease Association of America.
Regarding costs, Casgevy is priced at $2.2 million for a one-time treatment, while Lyfgenia costs $3.1 million. Treating everyone with these therapies will amount to billions. However, for those with insurance, coverage is assured. Some health experts argue that the cost is economical compared to the frequent hospitalizations sickle cell patients undergo over their lifetimes. Dr. Verdun emphasized the FDA's commitment to addressing access issues for all individuals, an aspect they will continue to work on.