The U.S. Food and Drug Administration (FDA) approved the first “cell-based gene therapy treatment” for use in individuals with beta-thalassemia who require regular blood cell transfusions. “Today’s approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research in an agency news release. “Given the potential health complications associated with this serious disease, this action highlights the FDA’s continued commitment to supporting development of innovative therapies for patients who have limited treatment options.” The therapy is a one-time single-dose treatment customized “using the patient’s own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin (a hemoglobin component)…The safety and effectiveness of [the gene therapy] were established in two multicenter clinical studies that included adult and pediatric patients with beta-thalassemia requiring regular transfusions. Effectiveness was established based on achievement of transfusion independence, which is attained when the patient maintains a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. Of 41 patients receiving [the therapy], 89 percent achieved transfusion independence. The most common adverse reactions associated with [the therapy] included reduced platelet and other blood cell levels…There is a potential risk of blood cancer associated with this treatment; however, no cases have been seen in studies of [the therapy].”
