FDA Approves First Gene Therapy for Beta-Thalassemia Patients Who Require Regular Red Blood Cell Transfusions: $2.8m Cost

Gene therapy-focused biotech venture bluebird bio announced recently that the U.S. Food and Drug Administration (FDA) approved ZYNTEGLO® (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy tailored to treat the underlying genetic cause of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. One of the world’s most expensive regimens, the price tag will equal $2.8 million for the potentially curative benefit across all ages and genotypes, aiming for the achievement of durable transfusion independence and normal or near normal hemoglobin levels. TrialSite points out a possibly competitive gene therapy product from San Rocco Therapeutics still in clinical development—and mired in litigation—that would be 4X less expensive and possibly superior, given the purported benefits of an insulator for this class of gene therapy.

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