The Food and Drug Administration has approved the world’s first medicine employing CRISPR gene-editing technology to treat sickle cell disease, giving thousands new hope to treat the painful disease.
“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,” said Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research.
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Two new treatments, Casgevy and Lyfgenia, were approved on Friday in a long-anticipated move that tackles genetic disorders by turning off or replacing specific genes.
Approximately 100,000 people in the United States are affected by sickle cell disease, which is the result of an inherited genetic mutation that produces a dysfunctional form of the protein hemoglobin, which carries oxygen in the bloodstream. Hemoglobin proteins in those affected with the disease are shaped in the form of a sickle, giving the disorder its name.
Casgevy targets the specific gene that causes the dysfunction, switching it off and allowing for the production of fetal hemoglobin proteins. Lyfgenia works by producing a gene-therapy-derived hemoglobin that functions similarly to the adult form of the protein.
Both products come from a patient’s blood stem cells, which are delivered in a one-time, single-dose infusion once they have been genetically modified. The treatments are approved for sickle cell disease patients over 12 years old with recurrent vaso-occlusive events.
“As we’ve seen throughout our history, when the U.S. government invests in innovation, we can achieve breakthroughs that would otherwise be impossible, and save and improve lives on a vast scale,” President Joe Biden in response to the announcement said.
Sickle cell disease occurs most frequently in black populations, affecting about 1 in every 365 black births in the United States. The disorder also occurs in 1 in every 16,300 Hispanic American births, according to the Centers for Disease Control and Prevention.
Sickle cell disease occurs mostly among descendants from parts of the world where malaria is endemic. Genetic carriers for sickle cell disease, also referred to as those with the sickle cell trait, have some protective advantages against malaria.
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, told reporters that he estimates that the treatment will be reserved for those with frequent hospitalizations due to severe complications from the disease, affecting approximately 20% of sickle cell disease patients.
“This is really for those whose lives have been significantly impacted, who sometimes look like they are going to suffer end organ damage from their sickle cell disease,” Marks said.
Verdun also indicated in a press briefing on Friday that both Casgevy and Lyfgenia have the potential for curative properties for the disease depending upon the results of long-term follow-up studies over the next 15 years.
“I think over time, in terms of getting to that place where we’re saying at what point [the treatments are] curative, we have to just follow [them] over time but … the results of both trials were quite encouraging,” Verdun said.
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Marks said that Friday’s announcement represents “an important medical advance” that will be vital in addressing other “potentially devastating diseases.”
“This significant medical advancement holds tremendous promise for developing additional life-saving treatments, and it gives hope to the millions of Americans who live with other rare diseases,” Biden said.
