Many drugs, whether they are indicated for common or rare diseases, come at very high prices. Zynteglo, an investigational gene therapy for beta thalassemia, is most likely to stick to this trend. It is currently awaiting a PDUFA review, which is slated for the 19th of August, 2022. Pending approval, along with positive data and the possibility to save patients money, may lead to a high cost for this gene therapy. According to an article published in Pharmaceutical Technology, Zynteglo may even be priced at $1 million.

About Beta Thalassemia

First thing’s first, let’s get a better understanding of what this rare disease is. Beta thalassemia is a blood disease caused by an issue with hemoglobin, which is a deficiency in this case. This disease occurs as three different types, which are minor, intermedia, and major. These types relate to the severity of the symptoms, with people who have minor beta thalassemia often being asymptomatic and those who have major needing lifelong medical care. This disease is caused by a mutation on the hemoglobin beta gene.

Those who have a mutation on one copy of this gene often experience minor beta thalassemia while those who have mutations on both copies experience the intermedia or major types. The symptoms of this disease include fatigue, weakness, shortness of breath, dizziness, headaches, blood clots, pallor, and splenomegaly, which is an enlargement of the spleen. Treatments for beta thalassemia depend on the type one has. Those with major require blood transfusions, which can be harmful if they cause iron overloads. If the spleen is heavily affected, surgery can be done to correct it. There is also an FDA approved treatment called Thiotepa.

Zynteglo and Its Pricing

European beta thalassemia patients have already experienced the astronomical price point of Zynteglo, as the European Medicines Agency (EMA) approved of the gene therapy back in 2019. When it became available to them, it was priced at EUR 1.6 million, which translates to 1.8 million in USD. However, this cost did not sit well with patients in Europe and resulted in pushback from the market.

This may lead to a more affordable cost off the bat for U.S. patients. However, we just don’t know yet. A representative from bluebird bio, the company developing Zynteglo, has said that no price has been set. We do know that there are multiple factors influencing this price, such as:

• The option of Zynteglo as a potential cure that only requires one-time administration
  • This awaits confirmation from long-term extension trials
• The value this one-time treatment provides to patients, especially those younger in age
  • It would remove the dependence patients have on blood transfusions
    • This could save patients as much as $100,000 annually
  • It would improve quality of life
• The price of research, development, and manufacturing, which is very high for gene therapies
• The actual FDA approval of the medication
• Medicaid funding due to the high number of beta thalassemia patients covered by Medicaid

All of these factors will play a role in the pricing of Zynteglo.

Diving Deeper into Long-Term Data

While we can’t yet know if Zynteglo is truly a one-time cure for beta thalassemia, bluebird is currently conducting a long-term observational study called LTF-303. The data from this study has been positive so far, with an update from March of 2021 stating that 40 of 51 participants had achieved transfusion independence.

Looking Forward

There’s no way to know the price of Zynteglo if and when it receives FDA approval, but it is not unlikely that the price will be high. All of these factors play a role, but hopefully, they will lead to an affordable and accessible option for beta thalassemia patients.