The latest price tag for a gene therapy, a treatment meant to provide a long-lasting effect with a single dose, is now set. The treatment, Zynteglo, developed for the rare blood disease beta thalassemia, will cost roughly $1.8 million.
Bluebird Bio (NASDAQ: BLUE), which just won approval of Zynteglo in Europe two weeks ago, announced the price Friday during the European Hematology Association’s (EHA) annual meeting in Amsterdam. The news makes Zynteglo the second-ever medicine to come to market with a list price more than a million dollars. The first, also a gene therapy, was the spinal muscular atrophy treatment Zolgensma, which was approved by the FDA last month. Its developer, Novartis (NYSE: NVS), priced the therapy at about $2.1 million.
The Zolgensma and Zynteglo approvals are crucial test cases for the economic viability of gene therapies. The therapeutic effects of these one-time treatments are intended to last for life, in contrast to traditional medicines that people take chronically. But those effects aren’t guaranteed. To this point, it’s unclear how long gene therapies will last, as their clinical track records are too short. Thus, developers and insurers are grappling with how to pay for them while accounting for the uncertainty.
In setting its price, Bluebird followed the playbook of Novartis, which is working on “outcomes-based agreements” with insurers that tie Zolgensma’s price tag to its performance. Novartis also said that it was talking with insurers about options to pay for the gene therapy over a period as long as five years.
In its EHA presentation, Bluebird said that the full price of Zynteglo could be spread out over five years, at approximately $357,567 annually. Payment models may vary by country, and the company is working with each country on the next steps to bring the gene therapy to the market. It vows not to boost the price of Zynteglo above changes in the consumer price index. Zynteglo could be priced within a “reasonably close range” in the US, should the FDA eventually approve it, according to a Reuters report.
In setting Zynteglo’s price, Bluebird points to the therapy’s potential to lower healthcare costs. Patients who have beta thalassemia are unable to produce sufficient amounts of hemoglobin, the oxygen-carrying protein in blood. Depending on the severity of their disorder, beta thalassemia patients may need blood transfusions every two to four weeks for the duration of their lives. The goal of Bluebird’s gene therapy is to reduce, or eliminate the need for such transfusions, ideally for life.
[The following two paragraphs added with analyst comments.] Zynteglo’s price is double the $900,000 estimate calculated by SVB Leerink. Mani Foroohar, an analyst for the firm, wrote in a research note that even though the Zynteglo and Zolgensma prices work out to be lower over time compared to currently available rare disease treatments, the Bluebird gene therapy is a transplant medicine product, which adds more costs to the healthcare system. In contrast, the Novartis gene therapy is a simple intravenous infusion, he wrote.
Foroohar also expressed concerns about the manufacturing of Zynteglo. Despite being in development for years, the Bluebird gene therapy won’t be available commercially until 2020 “due to lack of manufacturing readiness,” he wrote. Bluebird is making changes to its manufacturing process, and Foroohar said it’s unclear whether the Bluebird gene therapy that reaches the market will live up to what was seen in clinical trials.
“More concerning is that [Bluebird] was unable to execute on the manufacturing process development that is the critical core competency of any cell therapy franchise, perhaps raising questions on the company’s ability to hit stated timelines for many of its programs,” Foroohar wrote.
New long-term data presented at EHA showed that Bluebird’s therapy has freed a genetic subset of patients in an early stage trial from transfusions for a median of 3.8 years. European regulators granted a “conditional” approval to Bluebird a few weeks ago for that particular genetic subset of beta thalassemia patients.
The conditional approval means Bluebird must continue to provide additional data demonstrating the benefit of its therapy. Bluebird must also renew its gene therapy’s approval annually until it has enough data to convert the drug’s status to standard marketing authorization. Trials supporting a potential US approval, meanwhile, are ongoing. Bluebird aims to begin selling Zynteglo in the US in 2020. It’s also testing the drug in patients with sickle cell disease, another rare blood disorder.
Shares of Bluebird sank 3 percent, to about $120 apiece, in midday trading Friday.
基因治疗的最新价格标签，一种旨在提供单一剂量长期疗效的治疗方法，现在已经确定。这种治疗方法， Zyntello ，为罕见的血液病β地中海贫血开发，将花费大约180万美元。
两周前刚刚在欧洲获得 Zynteolo 批准的 Bluebird Bio 公司（ NASDAQ ： BLUE ）上周五在阿姆斯特丹举行的欧洲血液学协会（ EHA ）年会上宣布了这一价格。这一消息使 Zyntello 成为第二个上市药品，标价超过100万美元。第一个也是基因疗法，是脊髓性肌萎缩症治疗佐尔甘斯玛，上个月获得 FDA 批准。它的开发者诺华（Novartis）（ NYSE ： NVS ）给这种疗法定价约210万美元。
Zolgensma 和 Zyntello 批准是基因治疗经济可行性的关键测试案例。与人们长期服用的传统药物不同的是，这些一次性治疗的治疗效果旨在维持生命。但这些影响并不能保证。到目前为止，尚不清楚基因治疗将持续多久，因为他们的临床记录太短。因此，开发商和保险公司在考虑不确定性的同时，正努力解决如何为它们付费的问题。
Bluebird 在定价时遵循了诺华( Novartis )的策略，该公司正与保险公司签订“基于结果的协议”，将佐尔甘马的价格与业绩挂钩。诺华（Novartis）还表示，它正与保险公司讨论在长达五年的时间内支付基因治疗费用的选择。
Bluebird 在其 EHA 演示中表示， Zyntello 的全部价格可在五年内分摊，每年约357567美元。支付模式可能因国家而异，该公司正与各国合作，采取下一步措施将基因治疗推向市场。它承诺不会将 Zyntello 的价格推高至消费者价格指数的变动之上。路透( Reuters )的一份报告显示，如果 FDA 最终批准 Zyntello ， Zyntello 的定价可能在美国“相当接近的区间”。
在确定 Zyntello 的价格时， Bluebird 指出了这种疗法降低医疗成本的潜力。患有β-地中海贫血的患者不能产生足够数量的血红蛋白（血液中含氧的蛋白质）。根据其疾病的严重程度，β地中海贫血患者可能需要在其生命期间每两到四周输血一次。蓝鸟基因疗法的目标是减少或消除这种输血的需求，这是理想的生活。
Zyntello 的价格是 SVB Leelirink 估计的90万美元价格的两倍。该公司分析师 Mani Foroohar 在一份研究报告中写道，尽管 Zyntello 和 Zolgensma 的价格会随着时间的推移而降低，但 Bluebird 基因疗法是一种移植医学产品，它增加了医疗保健系统的成本。相反，他写道，诺华（Novartis）的基因疗法是一种简单的静脉输液。
Foroohar 还对 Zyntello 的生产表示关切。他写道，尽管蓝鸟基因疗法已经发展了多年，但由于“缺乏制造准备”，它要到2020年才能商业化。Bluebird 正在改变其制造工艺， Foroohar 说，目前还不清楚这种进入市场的 Bluebird 基因疗法是否能满足临床试验的要求。
Foroohar 写道：“更令人担心的是，（ Bluebird ）不能执行任何细胞治疗特许经营权的关键核心竞争力的制造工艺开发，这或许会令人质疑该公司能否在许多计划中达到规定的时间表。”
EHA 提供的新的长期数据显示， Bluebird 的治疗使早期试验中的患者从输血中释放出一个基因子集，平均时间为3.8年。几周前，欧洲监管机构批准 Bluebird 对β地中海贫血患者的特定基因子集进行“有条件”批准。
有条件的批准意味着蓝鸟必须继续提供额外的数据证明其治疗的好处。Bluebird 还必须每年更新其基因治疗的批准，直到有足够的数据将该药物的地位转化为标准的市场授权。与此同时，支持美国可能批准的试验正在进行中。蓝鸟计划在2020年开始在美国销售 Zyntello 。它还在对镰状细胞病（另一种罕见的血液病）患者的药物进行检测。
上周五午盘， Bluebird 股价下跌3%，至每股120美元左右。