After a manufacturing specification delay, Bluebird Bio has officially launched Zynteglo, its first gene therapy, andtapped 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, whos moving up to the global leadership team.

Heffron is tasked with charting the course for Bluebirds first commercial launch of its first product, Zynteglo, to treatbeta thalassemia, a rare inherited disease marked by reduced production of oxygen-carrying hemoglobin in red blood cells.

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And Heffron knows a thing or two about blood disorders. Before Bluebird, she headed up global marketing for Celgenes 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 Hospitalof Heidelberg serves as the drugs first qualified treatment center inthe country, and Bluebird said its working with institutions that have expertise in stem cell transplant as well as in treating patients with [beta thalassemia] to establish more centers.

RELATED:Bluebird Bio readies Zynteglo launch as EU approves 'refined' manufacturing

During a presentation at the annual J.P. Morgan Healthcare Conference on Tuesday, Bluebird CEO Nick Leschly confirmed the companysvalue-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 coverabout half of Germanys 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 facilitiesand contract partners for itslentiviral vector and drug product, Leschly said Tuesday.

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In the U.S., Bluebird has started itsrolling submission for approval and is in talks with the FDA regarding the requirements and timing of the various components of the application. Its 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 years American Society of Hematology annual meeting in December, none of the 17 patients enrolled in group Cwhich used an improved stem cell harvest technique and a new manufacturing processrequired regular blood transfusions post-treatment. Nine patients had beenfollowed for at least six months at that data cutoff.

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Bluebird Bio taps ex-Celgene exec Heffron to lead its first gene therapy launch - FiercePharma

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