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World’s Most Expensive Drug: FDA Approves $2.8 Million Cell-Based Gene Therapy In The US

Published in Financial.

The US Food and Drug Administration has now approved the most expensive drug in the nation at $2.8 million, reveals a Reuters report.

The approval was for Bluebird Bio’s gene therapy medication for patients suffering with a rare disorder requiring regular blood transfusions. The treatment is for patients suffering from beta-thalassemia which causes an oxygen shortage in the body and often leads to liver and heart issues.

There are around 1,500 patients in critical condition suffering from this disease and need blood transfusions every two to five weeks. The therapy that’s branded as ‘Zynteglo’ while lifesaving, can experience resistance from insurance companies for its hefty price tag.

Gene therapies have a reputation of being expensive since they’re mostly curative and they often experience difficulty in securing insurance coverage.

In 2019 when Novartis unveiled its $2.1 million therapy dubbed Zolgensma, insurers were hesitant and unwilling to cover the same, which forced the company to offer discounts while offering ‘outcome-based’ instalment payments for the therapy.

Bluebird calls Zynteglo as a one-time treatment that would no longer require the patient to need transfusions, helping save the patient’s money in the long run.