The Food and Drug Administration (FDA) recently announced its first-ever approved gene therapy in the United States, an article posted on the National Organization for Rare Disorders website said.
FDA approval was given to Kymriah to treat certain cases involving children and young adults who have cancer of the blood and bone marrow known as acute lymphoblastic leukemia (ALL). Kymriah will modify a patients’ own cells to target and destroy the leukemia cells, the article said.
According to the FDA, approximately 3,100 patients age 20 and younger are diagnosed with ALL each year.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadline cancer,” FDA Commissioner Scott Gottlieb said in the article.
Kymriah will be used for patients whose cancer has not responded to, or has returned after, traditional treatment, according to the FDA. In clinical trials, 83 percent of patients responded to Kymriah.
Kymriah, developed by Novartis Pharmaceuticals, has the potential for treating many rare diseases.
David Lebwohl, senior vice president and global head of cell and gene therapy at Novartis, said in the article the approval is “an important milestone in a long journey to transform clinical medicine using modern advances in genomics.”