Bluebird Bio Announces $1.8 Million Price Tag for Zynteglo Gene Therapy, Second Only to Zolgensma, Targeting β-Thalassemia

Arterial New Medicine (WeChat ID: biobeat1) has learned from foreign media that renowned biotechnology company Bluebird Bio recently announced the pricing for its gene therapy, Zynteglo. The treatment is priced at approximately $1.8 million, making it the second drug on the market with a list price exceeding $1 million.

It is reported that shortly after Zynteglo received approval from the European Union in early June, Bluebird Bio announced the drug’s price on the 14th of the same month at the European Hematology Association (EHA) annual meeting held in Amsterdam. Zynteglo is a gene therapy for the treatment of beta-thalassemia, a rare blood disorder, capable of providing durable therapeutic effects through a single-dose infusion. Currently, only Zolgensma, a gene therapy developed by Novartis, is priced higher than Zynteglo. Zolgensma, the first gene therapy approved for the treatment of spinal muscular atrophy, is priced at approximately $2.1 million.

Patients with β-thalassemia are unable to produce sufficient amounts of hemoglobin, the oxygen-carrying protein in blood. Depending on the severity of their condition, patients with β-thalassemia may require blood transfusions every two to four weeks. In setting the price for Zynteglo, bluebird bio considered that the therapy can reduce medical costs, eliminate the need for transfusions, and provide lifelong therapeutic benefits.

The approvals of Zolgensma and Zynteglo serve as key test cases for the economic viability of gene therapies. Unlike traditional medications that require long-term administration, these therapies offer a one-time treatment with sustained efficacy. However, due to the limited duration of clinical trial follow-up, it remains unclear exactly how long the therapeutic effects will last. Consequently, some pharmaceutical developers and insurers, acknowledging this uncertainty, are actively seeking solutions to address the issue.

When pricing Zynteglo, BlueBird Bio referenced Novartis. Novartis has linked the price of Zolgensma to its therapeutic efficacy. Furthermore, Novartis stated that it is discussing five-year payment plans for gene therapies with insurers and has reached “outcome-based agreements” with them.

Bluebird Bio stated that the full price of Zynteglo can be paid in installments over five years, with annual payments of approximately $357,567, and its payment model is adapted to local conditions. According to Reuters, if the drug receives final approval from the U.S. FDA, Zynteglo may be priced within a “reasonable” range in the United States, and the company also stated that it would not raise prices above the change in the Consumer Price Index. Currently, Bluebird Bio is collaborating with several countries to plan the market launch of the Zynteglo gene therapy.

Mani Foroohar, an analyst at Bluebird Bio, wrote in a research report that the prices of Zynteglo and Zolgensma will decrease over time. Compared with Novartis’s intravenous gene therapy, Bluebird’s gene therapy is a transplantable medical product, which increases the cost to the healthcare system.

Mani Foroohar expressed concern that, despite being in the development stage for many years, Bluebird Bio cannot guarantee the commercialization of Zynteglo by 2020 due to a lack of manufacturing readiness. More troublingly, Bluebird Bio’s inability to execute manufacturing process development has eroded its core competitiveness in the cell therapy franchise. This may lead to external skepticism about whether the company can complete its proposed projects within the specified timelines, and it remains unclear whether the market-launched Zynteglo gene therapy will achieve outcomes consistent with those observed in clinical trials.

It is worth noting that the European Union’s approval of Zynteglo a few weeks ago was also “conditional,” meaning that the company must continue to provide additional data to demonstrate the benefits of its therapy. Bluebird Bio must also submit data on this gene therapy to the relevant regulatory authorities annually until it has sufficient data to support the drug’s market authorization.

Currently, Bluebird Bio is testing the efficacy of Zynteglo in sickle cell disease and plans to begin selling the drug in the United States in 2020.

(Compiled by Wang Chan)