The European Medicines Agency (EMA) granted conditional approval to the autologous gene therapy, Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) in June, but bluebird bio quickly quashed hopes of a speedy launch when it revealed it was still talking to regulators about the manufacturing process.
In light of its then-ongoing efforts to narrow commercial drug product specifications, bluebird set a 2020 launch target for the gene therapy.
Now, bluebird has pulled forward its target and the company hopes to enroll its first commercial patient before the end of 2019.
Bluebird set the 2019 target after the EMA approved the refined Zynteglo commercial drug product manufacturing specifications.
The EMA approval clears one of the barriers to the commercial use of Zynteglo in Europe. However, as bluebird's chief commercial officer, Alison Finger, told investors at an event last month, the nature of Zynteglo makes the already challenging task of launching a drug in Europe harder still.
Finger said, “A lot of different planes have to land [before we treat patients], because it is about getting the qualified treatment centres online, signing the agreements with them, training them, getting everything set up for the manufacturing process, as well as the reimbursement and the changes in our release specs and the Type II variation that we announced in June.”
With the Type II variation now resolved, Bluebird can focus its energy on the other tasks that it must complete before enrolling and treating its first patient.
Readying European launch
The first patient is likely to be from Germany, as the reimbursement process in Germany will enable bluebird to start treating patients after entering into an agreement with a health insurer, which are known as a ‘sick funds’. The health technology assessment (HTA) process and agreement on price with the government regulated public health insurance system come later.
In France, Italy and the UK — the other countries targeted by bluebird — treatment can only begin after the HTA process is completed and a price is agreed. Talks are underway in all four countries and bluebird is publicly encouraged by how payers have responded to the staggered, outcomes-based payment model it has proposed to reduce the upfront cost of Zynteglo.
Finger said, “They do think the model we’ve put forward is a viable model for thinking about how they pay for gene therapy.”
The initial focus on Germany means use of Zynteglo will start close to where it is manufactured. Bluebird has tasked Apceth Biopharma, a German subsidiary of Hitachi Chemical, with producing the gene therapy for the European market.