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β地中海贫血基因疗法上市在即,定价可能高达210万美元

青瓦 青瓦 来源:医药魔方
2019-01-11
基因治疗
原文

正在召开的第37届JP摩根健康医疗投资年会上,Bluebird bio(蓝鸟生物)透露了其基因治疗药物LentiGlobin可能定价为210万美元。


LentiGlobin是一种用于治疗输血依赖性β地中海贫血(TDT)的基因疗法,通过慢病毒载体将表达正常血红蛋白β亚基的基因在体外植入到从患者体内取出的造血干细胞中,再将这些细胞回输到患者体内。


TDT是由β-珠蛋白基因突变引起的遗传性血液病,引发无效的红细胞生成,从而导致严重的贫血。TDT患者需要定期输血以维持血红蛋白(Hb)的存活,不过这种慢性输血容易提高铁超负荷的风险,进而可能导致多器官损害,并缩短预期寿命。目前全球约有30万人患有TDT,美国约1万人。


2018年12月3日,bluebird公布了Lentiglobin在TDT患者中两项III期研究Northstar-2和Northstar-3的新数据。


Northstar-2研究主要评估疗效,共招募16名(两名儿童和14名青少年/成人)非β0/β0基因型患者(年龄8-34岁),11名患者的随访时间在数据截止时至少为3个月,且有10名在最后一次随访时(治疗后3-18个月)停止接受输血。结果显示,患者开始产生基因治疗衍生的血红蛋白和接近正常的血红蛋白水平,并在大多数患者中消除了输血的需要。


Northstar-3研究主要评估安全性,招募了患有更严重的β0/β0基因型或IVS-I-110突变的患者,安全性处理措施与白消安清髓预处理方案一致,包括血管闭塞性肝病的严重不良事件(SAE)。该试验仅报告了1例可能与LentiGlobin有关的3级血小板减少症的SAE。


去年10月份,Bluebird已经向欧盟(EMA)递交了LentiGlobin治疗TDT和非-β0/β0基因型TDT青少年及成人患者的上市许可申请(MAA)。EMA已授予LentiGlobin治疗TDT的孤儿药资格、快速通道资格、优先药物资格(PRIME)。同时,该款产品也被FDA授予治疗TDT的孤儿药资格和突破性药物资格。


因此,从临床数据及快速通道的审评时间上看,这款疗法很可能会获得监管部门的批准。据外媒透露,LentiGlobin今年很可能在在欧洲获得批准,2020年有望在美国获得批准。随着药物的获批在即,bluebird计划将更多资源投入到定价和商业化方面。


“目前计划的浮动定价在210万美元左右,但也不排除低于这个价格的可能”,该公司首席执行官Nick Leschly在接受《华尔街日报》采访时坦言,“昂贵的定价是这种药物内在价值的真实反映,这不仅是生命质量的改善,更是生命长度的延伸。无论最终定价能否如期,我们都希望这款产品能够解决一些医疗问题。”


毫无疑问,210万美元的价格高得惊人。从这番定价中,我们也看到了基因细胞治疗价格一路飙升的趋势。比如Spark Therapeutics开发用于遗传性眼病的药物,每只眼睛治疗费用约为85万美元,诺华公司的白血病CAR-T药物Kymriah售价47.5万美元,其竞品吉利德的Yescarta售价为37.3万美元。 


如此昂贵的药价,如何让更多患者可及,亦是让华尔街投资者担忧的问题。Bluebird在大会上向投资者概述了其一项超过五年的支付计划。该支付与一些基因细胞治疗企业提出的“按疗效付费”类似,取决于基因疗法的持续有效性。例如Spark为患者购买保险,如果不起作用,患者可以获得赔偿,同时还向还向CMS提出一些支付计划方案。


Nick Leschly说Bluebird不是第一家考虑这种计划的公司,但显然是最先采用这种方案的公司之一。 一位企业CEO指出,随着基因细胞疗法的快速进展,大多数药物的定价都将超过100万美元,这也有望推动整个行业支付方式的变革。


JP摩根分析师Cory Kasimov听完Leschly的报告后总结道,“我们的目标是重生。这是一个伟大的声明,一个伟大的梦想,一个伟大的愿景。我们非常重视。”


Leschly还提到,Bluebird希望在2022年之前获批四种产品,此外公司还有很多临床前项目在推进。但就LentiGlobin定价问题,Leschly承认这也是他们将产品推向市场的策略之一。


Leschly在演讲中海反复强调,大家关注的重点应该是价值而不是价格,尽管治疗可能是昂贵的,但如果一切按计划进行,将使患者终身治愈。


参考资料:With Likely Approvals on the Way, Bluebird Bio Offers up Pricing Strategy for Gene Therapy

机器翻译
At the 37th JP Morgan Health Care Investment Annual Meeting, Bluebird Bio revealed that its gene therapy drug LentiGlobin may be priced at $2.1 million.

LentiGlobin is a gene therapy for the treatment of transfusion-dependent β-thalassemia (TDT) by implanting the gene expressing the normal hemoglobin β subunit in vitro via a lentiviral vector into hematopoietic stem cells removed from the patient and returning these cells to the patient.

TDT is an inherited blood disorder caused by mutations in the β-globin gene, triggering ineffective erythropoiesis, which leads to severe anemia.TDT patients require regular blood transfusions to maintain hemoglobin (Hb) survival, but this chronic transfusion can easily elevate the risk of iron overload, which in turn may lead to multiple organ damage and shorten life expectancy.At present, about 300,000 people worldwide suffer from TDT, and about 10,000 people in the United States.

On December 3, 2018, Bluebird published new data from two phase III studies of Lentiglobin in TDT patients, Northstar-2 and Northstar-3.

The Northstar-2 study assessed efficacy primarily, enrolling a total of 16 (two children and 14 adolescents/adults) patients with non-β0/β0 genotypes (age 8 – 34 years), 11 patients with a follow-up time of at least 3 months at the time of data cut-off, and 10 who discontinued receiving a blood transfusion at the time of last follow-up (3 – 18 months after treatment).The results showed that patients started to produce gene therapy-derived hemoglobin and near-normal hemoglobin levels and eliminated the need for blood transfusion in most patients.

The Northstar-3 study primarily assessed safety, enrolled patients with more severe β0/β0 genotypes or IVS-I-110 mutations, and the safety management measures were consistent with the busulfan myeloablative conditioning regimen, including serious adverse events (SAEs) of vascular occlusive liver disease.This trial reported only one SAE with grade 3 thrombocytopenia possibly related to LentiGlobin.

In October last year, Bluebird has submitted to the European Union (EMA) a marketing authorization application (MAA) for the treatment of TDT and non-β0/β0 genotype TDT in adolescent and adult patients.EMA has granted LentiGlobin Orphan Drug Qualification, Fast Track Qualification, Priority Drug Qualification (PRIME) for TDT.At the same time, the product was also granted orphan drug status and breakthrough drug status for TDT treatment by the FDA.

Therefore, from the perspective of clinical data and fast-track review time, this therapy is likely to be approved by regulatory authorities.According to foreign media, LentiGlobin is likely to be approved in Europe this year and is expected to be approved in the United States by 2020.Bluebird plans to invest more resources in pricing and commercialization as drug approvals are forthcoming.

"The current planned floating pricing is around US $2.1 million, but it does not rule out the possibility of below this price", the company's CEO Nick Leschly admitted in an interview with The Wall Street Journal that "expensive pricing is a true reflection of the intrinsic value of this drug, which is not only an improvement in the quality of life, but also an extension of the length of life.Regardless of whether the final pricing can be as scheduled, we hope that this product can solve some medical problems."

There is no doubt that the price of $2.1 million is surprisingly high.From this pricing, we also saw a trend of soaring prices for gene cell therapy.For example, Spark Therapeutics has developed drugs for hereditary eye diseases, which cost about $850,000 per eye, and Novartis' leukemia CAR-T drug Kymriah costs 47.For $50,000, its rival, Gilead's Yescarta, sells for 37. 5%.$30,000.

How to make more patients accessible at such expensive drug prices is also a concern for Wall Street investors.Bluebird outlined a payment plan for more than five years to investors at the conference.This payment is similar to the "pay-as-you-go" proposed by some gene cell therapy companies and depends on the continued effectiveness of gene therapy.For example, if Spark buys insurance for patients, if it does not work, patients can get compensation, and at the same time propose some payment plans to CMS.

Nick Leschly said Bluebird was not the first company to consider such a plan, but was clearly one of the first to adopt it.A company CEO pointed out that with the rapid progress of gene cell therapy, most drugs will be priced at more than $1 million, which is also expected to drive changes in the way the entire industry pays.

JP Morgan analyst Cory Kasimov concluded after listening to Leschly's report, "Our goal is rebirth.This is a great statement, a great dream, a great vision.We take it very seriously."

Leschly also mentioned that Bluebird hopes to approve four products by 2022, in addition to the company's many preclinical projects are advancing.But on the issue of Lenti Globin pricing, Leschly admits that this is also one of their strategies to bring products to market.

Leschly repeatedly stressed in the speech that the focus of everyone's attention should be on value rather than price, although treatment may be expensive, but if everything goes according to plan, it will make the patient cure for life.

References: With Likely Approvals on the Way, Bluebird Bio Offers Up Prating Strategy for Gene Therapy

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