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