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Groundbreaking genetic engineering therapy for sickle cell anemia disease approved by the US Food and Drug Administration

Posted: Sunday, December 10, 2023. 4:05 pm CST.

By Jose Luis Cruz: The U.S. Food and Drug Administration (FDA) has approved two innovative cell-based gene engineering therapies, Casgevy and Lyfgenia, for treating patients 12 years and older with sickle cell disease (SCD), more commonly known as sickle cell anemia. This marks a significant and groundbreaking milestone in genetic medicine, particularly with Casgevy being the first to employ a novel genome editing technology. The gene editing is done using the more precise CRISPR technology and its success in humans provides great hope for the treatment of other debilitating diseases such as cystic fibrosis and cancer.

Sickle cell disease, affecting roughly 100,000 Americans, is a group of inherited blood disorders. Primarily affecting people of African descent, and to a lesser extent Hispanics, SCD is characterized by a mutation in the hemoglobin of the patient. This mutation causes red blood cells to assume a sickle shape, leading to blockages in blood vessels and reduced oxygen delivery. The result is severe pain, organ damage, and potentially life-threatening complications.

Nicole Verdun, M.D., director of the FDA’s Office of Therapeutic Products, emphasized the significance of these therapies in addressing the critical needs of SCD patients. Casgevy, the first to utilize CRISPR/Cas9 genome editing technology, involves modifying patients’ hematopoietic stem cells. This editing boosts the production of fetal hemoglobin, reducing red blood cell sickling.

Lyfgenia employs a lentiviral vector for genetic modification, leading to the production of HbAT87Q, a hemoglobin variant that lessens the risk of sickling. Both therapies involve harvesting the patient’s own blood stem cells, modifying them, and reinfusing them after high-dose chemotherapy.

The FDA says “The efficacy of Casgevy was demonstrated in a trial where 93.5% of evaluable patients experienced freedom from severe vaso-occlusive crises for at least 12 months. Common side effects included low blood cell counts and mouth sores.”

Lyfgenia’s effectiveness was shown in a study where 88% of patients achieved complete resolution of vaso-occlusive events within a specific period. Similar side effects were observed, and a black box warning for hematologic malignancy is included in Lyfgenia’s labeling.

Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, hailed these approvals as a testament to the FDA’s dedication to advancing safe and effective treatments for severe diseases. The FDA granted Priority Review, Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy designations to both applications. Casgevy is approved for Vertex Pharmaceuticals Inc., and Lyfgenia for Bluebird Bio Inc.

This breakthrough in gene therapy offers new hope to those suffering from sickle cell disease. By targeting the genetic root of the disease, Casgevy and Lyfgenia represent a significant leap forward in personalized medicine and the ongoing battle against genetic disorders.

The FDA, as part of the U.S. Department of Health and Human Services, ensures public health through the regulation of medical drugs, devices, and other health-related products. Yesterday’s approval of Casgevy and Lyfgenia highlights the agency’s role in fostering innovative treatments for complex health challenges.


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