FDA Approves $2.8M Gene Therapy for Patients with Beta-Thalassemia

The US Food and Drug Administration (FDA) approved Zynteglo, the first cell-based gene therapy for patients with beta-thalassemia. The treatment costs $2.8M and is the most expensive drug in the US; however, its developer promised up to 80% reimbursement of the cost if the therapy is ineffective.

The FDA approved Zynteglo (betibeglogene autotemcel) for treating adult and pediatric patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

Zynteglo is a one-time gene therapy product administered as a single dose created using the patient’s bone marrow cells that are genetically modified to produce functional beta-globin, a hemoglobin component.

“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, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research in a press 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.”

Beta-thalassemia is a type of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells in the blood through mutations in the beta-globin subunit, leading to insufficient delivery of oxygen in the body. The reduced levels of red blood cells can lead to a number of health issues, including dizziness, weakness, fatigue, bone abnormalities, and more severe complications.

Transfusion-dependent beta-thalassemia, the most severe form of the condition, generally requires life-long red blood cell transfusions as the standard course of treatment. However, these regular transfusions can be associated with multiple health complications of their own, including problems in the heart, liver, and other organs due to an excessive build-up of iron in the body.

The effectiveness of Zynteglo was established based on the achievement of transfusion independence, which is attained when the patient maintains a pre-determined hemoglobin level without needing any red blood cell transfusions for at least 12 months. Of 41 patients receiving Zynteglo, 89% achieved transfusion independence.

Late-stage negotiations

Bluebird Bio, the developer of Zynteglo, set the wholesale acquisition cost of the therapy at $2.8M “in recognition of its robust and sustained clinical benefit demonstrated in clinical studies.” The company says the treatment will alleviate the lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia, that can reach up to $6.4 million in the US.

The company offers an outcomes-based contract that includes a single upfront payment and up to 80% reimbursement of the cost of the therapy to contracted commercial and government payers if a patient fails to achieve and maintain transfusion independence up to two years following infusion.

As 70-75% of patients with transfusion-dependent beta-thalassemia are covered by commercial insurance, Bluebird Bio says it is “in late-stage negotiations” with leading commercial payers “with the potential to represent dozens of plans.” In addition, the company says it is engaging with state Medicaid agencies representing approximately 80% of publicly insured thalassemia patients.


1. FDA. FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions.

2. Bluebird Bio. bluebird bio Announces U.S. Commercial Infrastructure to Enable Patient Access to ZYNTEGLO®, the First and Only FDA-Approved Gene Therapy for People with Beta-Thalassemia Who Require Regular Red Blood Cell Transfusions.

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