WHO

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.

Zynteglo helped about 89% of people avoid blood transfusions over a 12-month period in clinical trials. Some of the most common side effects were mouth sores, fever, and vomiting.

Bluebird CEO, Tom Klima, in a conversation with Reuters revealed that the average cost of transfusions over the lifetime can be around $6.4 million. "We feel the prices we are considering still bring a significant value to patients," said Klima.

He added, talking about conversations with insurers regarding the one-time payment of the treatment, "Potentially, up to 80% of that payment will be reimbursed if a patient does not achieve transfusion independence, they (insurers) are very excited about that."

_{The FDA warned of a potential risk of blood cancer with the treatment but noted studies had no such cases.}

_{Bluebird expects to start the treatment process for patients in the fourth quarter. No revenue is, however, expected from the therapy in 2022 as the treatment cycle would take an average of 70 to 90 days from initial cell collection to final transfusion.}

What is Zynteglo?

Zynteglo is a gene therapy medication that treats kids and adults living with transfusion-dependent (severe) beta-thalassemia.

If you have this type of thalassemia, your body has trouble making hemoglobin — a protein that carries oxygen in your red blood cells. This leads to a lack of red blood cells, and oxygen has trouble traveling around your body. Mutations in one or more hemoglobin genes cause this, so Zynteglo is designed to replace mutated cells with normal ones. It aims to fix the problem at its source.

When Zynteglo was put up for approval, the FDA made it a top item on its to-do list. It helped Zynteglo get through the approval process faster by giving it priority review, fast track, and breakthrough therapy designations. The agency also gave it orphan drug status (a designation given to medications that treat rare medical conditions). For comparison, most new medications are approved through a standard review process.

How does Zynteglo work?

Zynteglo is a type of cell-based gene therapy. Bone marrow stem cells are taken from your body, genetically modified, and placed back into your body as a form of treatment.

However, a few steps go into this. If your care team decides you’re eligible for Zynteglo, this is what the general process of receiving it looks like:

To start, you’ll receive a couple medications that boost your stem cell numbers. Once there’s enough of these cells, they’ll be collected from your body in a process called apheresis.
After enough of these cells are collected, they’ll be sent to a lab. If not enough are collected the first time around, you’ll receive more rounds of medication and apheresis until enough are gathered.
At a lab, your stem cells will be genetically modified to create your dose of Zynteglo. Then, the final product will be sent back to your treatment center.
Once your Zynteglo dose is ready to go, you’ll receive a chemotherapy medication like busulfan daily for a few days. This gets rid of any remaining stem cells in your body. Zynteglo will replace these cells with genetically modified ones. As a precaution, you may also receive medications to prevent side effects, such as seizures and liver problems.
After at least 2 days have passed since your last dose of busulfan, you should be ready to receive Zynteglo.
After your infusion, you’ll stay in a hospital for about 3 to 6 weeks for monitoring purposes.

For more in the world of technology and science, keep reading Indiatimes.com.

Highlights

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.