The $2.8 million gene therapy treatment is the most expensive US drug ever

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A unique gene therapy treatment recently approved by the US FDA is attracting attention not only for its clinical value, but also for its price: at $2.8 million, Bluebird Bio’s Zynteglo is the world’s most expensive drug. history of the United States.

Zynteglo is a treatment designed to treat the rare disease known as transfusion-dependent beta-thalassemia (TDBT), a blood disorder that reduces the number of hemoglobin and red blood cells in the blood, leading to a reduction in oxygen supply. Each dose is tailored to the individual’s genetic profile for maximum effectiveness.

Transfusion-dependent patients should undergo red blood cell transfusions as frequently as every two weeks. According to Bluebird, clinical trials of Zynteglo resulted in 89% of patients achieving “transfusion independence,” defined as not needing a transfusion for at least 12 months.

On Zynteglo’s price, Bluebird said $2.8 million was actually a deal because “the lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia can reach up to 6.4 millions of dollars”.

According to the FDA statement on Zynteglo’s approval, the drug regulator fast-tracked the drug through a pediatric disease voucher. TDBT is an inherited condition that dramatically shortens life expectancy, with the average American patient suffering from this disease dying at age 37. Bluebird estimates that between 1,300 and 1,500 people in the United States suffer from the disease.

TDBT is caused by genetic mutations, which gene therapies are designed to target, repair or replace, and return cells to normal functioning. Zynteglo works by adding functional, modified copies of the gene that causes TDBT to a patient’s bloodstream, a process Bluebird calls “one time” but can still take months to administer.

Bluebird said the drug will be available from the fourth quarter of 2022.

The most expensive medicine of all time

At $2.8 million for a treatment, Zynteglo dethroned the previously most expensive drug, another gene therapy product called Zolgensma priced at $2.1 million.

According to Reuters, gene therapies are often curative and their price is high, making them difficult for insurance companies to sell. In the case of Zolgensma, produced by Texas-based Novartis Gene Therapies, the drug was too expensive for many insurers, leaving patients without access to the drug.

Bluebird, which said it priced Zynteglo so high “in recognition of its strong and long-lasting clinical benefits,” at least appears to be aware of a possible backlash by putting a multimillion-dollar price tag on a potentially life-saving drug, come out early with plans to help fund treatment.

According to Bluebird, the company is working with commercial and government payers to establish upfront payment plans with an outcome-based agreement that will allow companies to reimburse 80% of the cost of therapy if the treatment does not work. So it’s good, then.

Bluebird said it was in “late-stage negotiations with major commercial payors” who could make it available on “dozens of plans.” The company said it is also working with state Medicaid agencies, which account for 80% of publicly insured TDBT patients, to ensure they have access to it as well.

Arguing that such an expensive drug is actually a good deal, which Bluebird claimed, may not be the best approach. Despite the life-saving nature of Novartis’ gene therapy drug, insurers simply wouldn’t pay, forcing the company to negotiate discounts similar to those Bluebird offers upfront, likely in an effort to avoid such a conversation. ®

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