Modern medicine is one step closer to potentially curing sickle cell disease.
On the heels of the U.K.’s authorizing the world’s first gene-edited treatment for sickle cell disease on Nov. 16, the U.S. Food and Drug Administration (FDA) approved the same treatment on Dec. 8.
“CASGEVY’s approval by the FDA is momentous,” Reshma Kewalramani, M.D., chief executive officer and president of Vertex Pharmaceuticals in Boston, said in a statement provided to Fox News Digital.
“It is the first CRISPR-based gene-editing therapy to be approved in the U.S.”
“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, in the agency’s press release.
“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval,” he also said “reflecting the FDA’s commitment to facilitating [the] development of safe and effective treatments for conditions with severe impacts on human health.”
The CASEVY treatment — also known as exa-cel, or exagamglogene autotemcel — uses technology that “edits” the genes that cause sickle cell disease.
To develop the drug, Boston-based Vertex and CRISPR Therapeutics, which is based in Switzerland, used a gene-editing system known as CRISPR-Cas9 to turn genes on and off, according to the Sickle Cell Disease Association of America website.
“CASGEVY is a first-in-class treatment that offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease,” said Kewalramani.
The inventors of the CRISPR technology won the Nobel Prize in 2020.
CRISPR stands for “clustered regularly interspaced short palindromic repeats,” which are repetitive sequences of DNA.
“The CRISPR technology [that is] being used with this treatment for sickle cell disease — a devastating inherited illness that profoundly affects survival and quality of life — is remarkable,” Dr. Mikkael Sekeres, chief of the division of hematology of Sylvester Cancer Center at the University of Miami, told Fox News Digital.
He was not involved in the drug’s development.
“The data from the admittedly small study in support of the treatment’s approval by the FDA are impressive, and the therapy was thought safe by a panel of independent experts,” Sekeres added.
The treatment is approved for people 12 and older with severe sickle cell disease who get recurrent pain syndromes known as vaso-occlusive crises, in which blood flow is blocked due to sickle cells.
The FDA also approved Lyfgenia, the first cell-based gene therapy for the treatment of sickle cell disease in people 12 years of age and older, per the agency’s press release.
What to know about sickle cell disease
Sickle cell disease is the most common inherited blood disorder in the United States, affecting an estimated 100,000 Americans, as the National Institutes of Health (NIH) noted on its website.
It disproportionately affects people of Black or African ancestry.
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