After a manufacturing specification delay, Bluebird Bio has officially launched Zynteglo, its first gene therapy, and tapped a new leader to run the operation.
Nicola Heffron, a former exec with Celgene, Shire and GlaxoSmithKline, has joined Bluebird as its European chief, Bloomberg reported, just as the company gets its rollout underway there. She is replacing Andrew Obenshain, who’s moving up to the global leadership team.
Heffron is tasked with charting the course for Bluebird’s first commercial launch of its first product, Zynteglo, to treat beta thalassemia, a rare inherited disease marked by reduced production of oxygen-carrying hemoglobin in red blood cells.
And Heffron knows a thing or two about blood disorders. Before Bluebird, she headed up global marketing for Celgene’s myeloid portfolio, her LinkedIn profile shows.
Wednesday, Bluebird revealed that the $1.76 million-per-treatment gene therapy is now available in Germany. The University Hospital of Heidelberg serves as the drug’s first qualified treatment center in the country, and Bluebird said it’s “working with institutions that have expertise in stem cell transplant as well as in treating patients with [beta thalassemia]” to establish more centers.
During a presentation at the annual J.P. Morgan Healthcare Conference on Tuesday, Bluebird CEO Nick Leschly confirmed the company’s value-based payment model for the costly onetime therapy. Under that arrangement, payments of €315,000 ($351,000) each are made in five installments over five years. Except for the first round of expenses, payers only pay the rest if Zynteglo delivers on its therapeutic promise.
So far, the reimbursement agreements Bluebird has penned using that model can cover about half of Germany’s patients, according to Leschly.
Novartis has also rolled out a similar program for its $2.1 million spinal muscular atrophy gene therapy Zolgensma.
Bluebird won its European nod for Zynteglo last year but delayed the launch after a manufacturing specification hiccup. The Cambridge, Massachusetts-based biotech has established a manufacturing network that includes both internal facilities and contract partners for its lentiviral vector and drug product, Leschly said Tuesday.
In the U.S., Bluebird has started its rolling submission for approval and is in talks with the FDA “regarding the requirements and timing of the various components” of the application. It’s expecting to finish the process in the first half of 2020.
Outside of beta thalassemia, Bluebird is testing the same drug, also known as LentiGlobin, in sickle cell disease, which is also marked by an abnormality in hemoglobin.
According to phase 1/2 data presented at last year’s American Society of Hematology annual meeting in December, none of the 17 patients enrolled in group C—which used an improved stem cell harvest technique and a new manufacturing process—required regular blood transfusions post-treatment. Nine patients had been followed for at least six months at that data cutoff.