US approves world's most expensive drug

The US Food and Drug Administration has approved bluebird bio's gene therapy for patients with a rare disorder requiring regular blood transfusions, and the drugmaker priced it at a record $2.8 million, Report informs referring to Reuters.

The approval sent the company's shares 8% higher and is for the treatment of beta-thalassemia, which causes an oxygen shortage in the body and often leads to liver and heart issues.

The sickest patients, estimated to be up to 1,500 in the United States, need blood transfusions every two to five weeks.

The therapy, to be branded as Zynteglo, is expected to face some resistance from insurers due to its steep price, analysts say.

Gene therapies usually come with a high price tag as they are often curative and have faced hurdles in securing insurance coverage.

For instance, Novartis was in 2019 forced to offer discounts and work out "outcome-based" installments payments for its $2.1 million therapy Zolgensma after insurers balked at the drug's price.

Bluebird has pitched Zynteglo as a potential one-time treatment that could do away with the need for transfusions, resulting in savings for patients over the long term.

The average cost of transfusions over the lifetime can be $6.4 million, Chief Operating Officer Tom Klima told Reuters before the approval. "We feel the prices we are considering still bring a significant value to patients."

Bluebird has been in talks with insurers about a one-time payment option.