Aug 18, 2022 - Health

FDA approves $2.8 million gene therapy

A laboratory staff works at a non-profit gene therapy organization.
Photo: Eric Piermont/AFP via Getty Images

The FDA on Wednesday approved Zynteglo, a gene therapy from Bluebird Bio that treats patients with the blood disorder beta-thalassemia.

Why it matters: The one-time treatment — which helps patients who depend on regular blood transfusions and face decreased life expectancy — comes at a record price of $2.8 million.

  • It's only the third gene therapy approved by the FDA.

Details: Bluebird estimates there are between 1,300 and 1,500 people with beta-thalassemia, also known as beti-cel.

  • The company plans to use an outcome-based agreement, saying it'll reimburse insurers up to 80% of the cost of the therapy for any patients who still need regular transfusions after their treatment.

What they're saying: The Institute for Clinical and Economic Review, a group that evaluates drug pricing and effectiveness, said in a statement the drug has high long-term value, even with a price tag over $2 million.

The bottom line: Gene therapies offer a particular source of promise for treating rare diseases. But they've proved costly so far and the growing pipeline of such treatments could lead to staggering health care spending.

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