Bluebird Bio's gene therapy is priced second only to Zolgensma

, a well-known biotech company, Bluebird Bio, recently announced the price of its gene therapy, Zynteglo, for about $1.8 million, making it the second drug on the market to sell for more than $1 million,said.Zynteglo is understood to have just received EU approval in early June, when Bluebird Announcy announced the price of the drug at the annual meeting of the European Society of Hematology (EHA) in Amsterdam on the 14th of the same month. Zynteglo is a gene therapy used to treat β rare blood disease, thalassemia, and maintains lasting therapeutic effects through single-dose injections. Currently, only Novarma-developed Zolgensma gene therapy is more expensive than Zynteglo. Zolgensma is the first gene therapy to treat spinal muscular dystrophy, priced at about $2.1 million.Patients β with thalassemia are unable to produce sufficient amounts of hemoglobin, or oxygenated protein in the blood. Depending on the severity of the condition, β with thalassemia may need blood transfusions every two to four weeks. When setting Zynteglo's price, Bluebird Bio considered that the treatment could reduce medical costs, eliminate the need for blood transfusions, and keep the treatment effective for life.Zolgensma and Zynteglo approval is a key test case for the economic viability of gene therapy. Compared to traditional drugs that people take for a long time, these drugs can be treated once and maintain long-term efficacy. At present, however, due to the limited length of clinical trials, it is not clear how long gene therapy efficacy can be sustained. As a result, some drug developers and insurance companies are considering the instability of the incident and are trying to find a way to address the problem.Bluebird Creatures referred to Novaral when pricing Zynteglo. Novarma linked Zolgensma's price to its efficacy. In addition, Novart said it was discussing with insurers options to pay for gene therapy over the next five years and had reached a "results-based agreement" with them.Bluebird Said Zynteglo's full payment could be made in installments over a five-year period, at about $357,567 a year, and its payment model would be locally appropriate. If the drug is finally approved by the FDA, Zynteglo could be priced within a "reasonable" range in the U.S., Reuters reported, and the company said it would not raise prices above changes in the consumer price index. Bluebird Creatures is currently working with a number of countries to bring Zynteglo gene therapy to market.The prices of Zynteglo and Zolgensma will decrease over time, Bluebird Bio analyst Mani Foroohar wrote in a research note. Bluebird's gene therapy is a portable medical product that increases the cost of the health care system compared to Novartic gene therapy, which is administered intravenously.Mani Foroohar worries that although bluebird creatures have been in development for years, a lack of manufacturing preparations is not yet guaranteed to commercialize Zynteglo by 2020. More worryingly, Bluebird creatures lack the core competencies of cell therapy franchises because they are unable to perform manufacturing process development. This could lead to questions about the company's ability to complete the proposed projects within the specified time node, and it is unclear whether the Zynteglo gene therapy on the market will meet what clinical trials have seen.It is worth noting that the EU'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 treatment. Bluebird Must also provide annual data on the gene therapy to the relevant auditing authorities until it has enough data to support the drug's launch.Bluebird is currently testing Zynteglo's efficacy in sickle anemia and plans to begin selling the drug in the United States in 2020. (Arterial mesh)