Bluebird, cleared for Zynteglo launch, aims to dose first patients soon
Dogged for months by concerns over manufacturing, Bluebird Bio's ultra-pricey flagship gene therapy Zynteglo was finally cleared for launch in Germany just weeks ago. Now, with a manufacturing green light in hand, Bluebird is hoping to make good on its commercial hopes in the coming months.
Bluebird plans to dose its first commercial patients for Zynteglo in Germany in the first half of the year, the drugmaker said Tuesday.
In late January, Bluebird said the European Medicines Agency approved the “refined commercial drug product manufacturing specifications for Zynteglo," giving the drug the all-clear to launch in Germany. Bluebird has yet to seek FDA approval for the treatment.
Zynteglo, approved as a one-time treatment for patients 12 and over with transfusion-dependent beta-thalassemia, is Bluebird's first approved drug, though the CAR-T prospect it shares with Bristol-Myers Squibb, Ide-cel, is inching toward the finish line. The drugmaker pulled in $10 million in the fourth quarter in license and royalty revenues from that collaboration.
After scoring an approval last June, Bluebird said it would delay its launch for at least six months on manufacturing concerns. Regulators wanted the company to "tighten up" its manufacturing before treating its first commercial patient. But Bluebird put the extra time to good use, using it to qualify and train the staff and facilities that will handle the treatments.
Manufacturing has been a common concern for commercial gene therapies due to the exacting procedures needed to genetically code a patient’s cells. The processes require that cells be taken from a patient, cryopreserved, and shipped to a facility where they are genetically reprogrammed and new cells are manufactured in the lab. They are then shipped back for infusion into the patient, all in the shortest timeframe possible.
Bluebird has priced Zynteglo in Europe at €1.575 million ($1.77 million), a cost that is amortized over five years and paid only if it works on patients treated for transfusion-dependent beta-thalassemia. Bluebird previously said it planned to file its FDA application for Zynteglo by year-end 2019, but it has yet to send the therapy to U.S. regulators.
The drug is being manufactured in Europe by Apceth Biopharma, whose CEO Christine Guenther said the CDMO was “in the final stages of preparing to manufacture a cell-based gene therapy for commercial use.”
Feb 19, 2020
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