This New $2.8-Million Gene Therapy Is Now The Most Expensive Medicine In History

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapy ever. It is a one-off treatment for patients with a rare genetic blood disease. The single treatment will cost US$2.8 million. This makes it the most expensive medicine in history.

This gene therapy is called Zynteglo and is crafted to treat beta-thalassemia which is a rare disease with patients often requiring life-long blood transfusions. The gene therapy will be specific to every patient.

Bone marrow stem cells are harvested from the patient and then genetically modified to insert working copies of a gene that can effectively produce functional red blood cells. The modified stem cells are then infused back into the patient, with the positive effects of the one-off treatment that are expected to last a lifetime.

Clinical trials have found that 89% of patients become transfusion-independent after the treatment. However, this has its risks.

In approving Zynteglo the FDA noted a potential risk for cancer and recommends monitoring patients for 15 years after treatment with the gene therapy.

The approval of Zynteglo is somewhat historic as it’s only the third gene therapy to reach the market in the United States. It is also the first cell-based gene therapy to ever be approved in the US, meaning it’s the first authorized therapy where stem cells are removed, genetically modified, and then returned to a patient.

The company that is launching the medicine is bluebird bio.

“The lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia can reach up to $6.4 million in the U.S. and the average total health care cost per patient per year is 23 times higher than the general population,” the company explained in a statement. “bluebird estimates that there are approximately 1,300-1,500 individuals with transfusion-dependent beta-thalassemia in the U.S.”

Zolgensma’s $2 million price tag has been the source of extensive negotiation with health insurers over the past couple of years. Its manufacturer Novartis has tried to break its cost down into annual installments for insurers, spreading the price over three or four years.

Zynteglo was initially approved for use in the European Union back in 2019 but they had to settle things with health insurers over the treatment’s cost.