New Gene Therapy Becomes Most Expensive Drug Ever at $2.8M

The FDA has approved a gene therapy that's now the most expensive drug ever. Here's what we know about the drug and it's really high price tag.

Rachel Curry - Author

Aug. 19 2022, Published 11:59 a.m. ET

The U.S. Food and Drug Administration (FDA) has approved a gene therapy that's now the most expensive drug ever. Bluebird Bio’s Zynteglo costs a staggering $2.8 million.

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What is Zynteglo and why is it so expensive? Here are the details on the most expensive drug to ever hit the market.

The FDA approved Bluebird Bio’s gene therapy drug that costs $2.8 million.

Massachusetts-based gene therapy company Bluebird Bio Inc. (BLUE) just got through a major hurdle with the FDA approving its latest drug. Zynteglo costs $2.8 million, which makes it the most expensive drug yet.

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According to an FDA press release, Zynteglo (or betibeglogene autotemcel) treats “adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.”

The director of the FDA’s Center for Biologics Evaluation and Research, Peter Marks, M.D., Ph.D., said the approval “is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions.” He also said, “Given the potential health complications associated with this serious disease, this action highlights the FDA’s continued commitment to supporting the development of innovative therapies for patients who have limited treatment options.”

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How Zynteglo works.

Zynteglo treats beta-thalassemia, an inherited blood disorder that lowers hemoglobin and red blood cells in the blood by mutating the beta-globin subunit. The condition leads to insufficient oxygen in the body. People with beta-thalassemia are transfusion-dependent.

The FDA says about the drug, “Zynteglo is a one-time gene therapy product administered as a single dose. Each dose of Zynteglo is a customized treatment created using the patient’s own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin (a hemoglobin component).”

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Zynteglo shows success in helping patients become independent of transfusions.

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What makes Bluebird Bio’s Zynteglo so expensive?

For many, a $2.8 million price tag isn't attainable, but Zynteglo is only the third gene therapy drug the FDA has ever approved. According to the Institute for Clinical and Economic Review, the drug produces “high long-term value for the money.”

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Bluebird says transfusion-dependent beta-thalassemia patients require red blood cell transfusions every 2–5 weeks. Through the process, they can lose decades off their life. The lifetime medical care for someone with this condition can easily reach $6.4 million in the U.S. Bluebird wrote in a blog post, “The average total health care cost per patient per year is 23 times higher than the general population. Bluebird estimates that there are approximately 1,300–1,500 individuals with transfusion-dependent beta-thalassemia in the U.S.”

Bluebird Bio has stated it will reimburse 80 percent of the cost of the $2.8 million drug if it fails to provide transfusion independence.

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BLUE stock jumps on the news, but remains volatile.

Investors are taking a liking to Bluebird Bio stock in response to the recent FDA approval. The shares jumped 26.5 percent on Aug. 17 when the FDA first announced its approval. The shares have since cooled, but the stock is up 6 percent on Aug. 19, which suggests ongoing momentum.

BLUE stock is up 20.35 percent in the month ending Aug. 19 but remains in the red on a YTD basis at -45.61 percent.


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