The Food and Drug Administration on Friday approved two breakthrough treatments using the gene-editing tool CRISPR, a Nobel Prize-winning medical technology.
The first therapy, Casgevy, was developed by Vertex Pharmaceuticals and CRISPR Therapeutics and will be used to treat sickle cell disease, a type of anemia, according to data from the Centers for Disease Control and Prevention, that affects 100,000 Americans, most of whom are Black. Lyfgenia, a second gene treatment for sickle cell, was developed by bluebird bio, Inc.
“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, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, said in a statement.
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“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”
Sickle cell disease causes a shortage of red blood cells and can lead to severe pain, infection, acute chest syndrome, and even stroke. The only previously known cure for sickle cell disease was a bone marrow transplant, which risks adverse reactions by the patient’s immune system and presents difficulties in finding the right donor.
“It’s a game-changer,” Dr. Asmaa Ferdjallah, a pediatric hematologist at the Mayo Clinic in Rochester, Minnesota, told NBC News. “To really reimagine and rediscuss sickle cell disease as a curable disease and not as this painful and debilitating chronic disease is hope enough with this news.”
The Wall Street Journal reported that Vertex will manufacture Casgevy and will set a market price soon. The Institute for Clinical and Economic Review estimates that it may cost $2 million per patient.
Read it at The Wall Street Journal
