A manufacturing hiccup that Bluebird Bio’s believed might delay its gene therapy launch until 2020 has not only been overcome, the company now says it expects to enroll its first patients there yet this year.
The Cambridge, Massachusetts-based company Tuesday said the European Medicines Agency has approved the “refined commercial drug product manufacturing specifications for Zynteglo." Bluebird has yet to seek FDA approval for the treatment.
Bluebird Bio in June said the launch of the one-time treatment for patients 12 and over with transfusion-dependent beta-thalassemia might be delayed in the EU until 2020. Even though it was approved, regulators wanted Bluebird to "tighten up" its manufacturing before treating its first commercial patient. It said at the time that as it tweaked its manufacturing it also would use the time to qualify and train the staff and facilities that will handle the treatments.
Tuesday, Bluebird Bio Chief Commercial Officer Alison Finger said its goal is now to enroll “our first commercial patient in 2019.”
She said it was the first step in a country-by-country launch, to serve “TDT patients with Zynteglo and providing a treatment option that offers the possibility of a transfusion-free future.”
Bluebird Bio has priced Zynteglo in Europe at €1.575 million ($1.77 million), a cost that is amortized over five years and only if it works on patients treated for transfusion-dependent beta-thalassemia. Bluebird has yet to seek approval of the gene therapy in the U.S. but has told investors to expect it will file with the FDA by the end of this year.
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.”