- Germany-based Apceth Biopharma will be the commercial supplier of Bluebird bio's gene therapy Zynteglo in Europe, where it received approval earlier this week.
- European regulators cleared Zynteglo for certain patients with a rare blood disorder known as transfusion-dependent beta-thalassemia. Specifically, patients must be 12 years or older, have a particular genotype, and be able to receive a hematopoietic stem cell transplant but not have an appropriate stem cell donor.
- An estimated 2,000 to 3,000 patients in the European Union fit those criteria and could initially receive Zynteglo.
Apceth was one of the first contract manufacturers to work with Bluebird; as of late February, it remained the sole provider of the drug product supporting Zynteglo's commercial launch in Europe.
With approval there now secured, Apceth is tasked with ensuring there's enough Zynteglo to meet market demand. That may not be a huge problem early on, given that Bluebird expects a slow commercial build up. The number of patients eligible to receive the therapy is also relatively small compared to other medicines.
Still, manufacturing gene and cell therapies remains a notoriously expensive and complex process. Novartis, one of the world's largest drug companies, has reported troubles with the supply chain built around its CAR-T cell therapy Kymriah (tisagenlecleucel).
Apceth seems aware of the challenge.
"Being one of the very few companies worldwide to manufacture a cell-based gene therapy for commercial use marks a milestone for our company," CEO Christine Guenther said in a June 4 statement.
Backing Apceth in that milestone is its new owner, Hitachi Chemical.
Historically, Hitachi Chemical has focused more on producing functional materials and systems for products like electronics or energy storage devices. Yet the company has also been building out its business in the life sciences, with one example being the $75 million used in 2017 to purchase an ownership stake in PCT, a cell therapy developer and manufacturer.
About a year after that deal, Daiichi Sankyo tapped Hitachi Chemical for the manufacturing of its experimental regenerative medicines.
And by January of 2019, Hitachi agreed to buy Apceth for roughly $86 million. The deal completed in early April.
Bluebird has also been investing in the space, opening up earlier this year its first wholly owned manufacturing facility in North Carolina. In addition to Zynteglo, the company has several gene therapy candidates in mid- to late-stage testing, including Lenti-D for cerebral adrenoleukodystrophy and bb2121 for multiple myeloma.
With Zynteglo, Bluebird uses an inactive virus to give beta-thalessemia patients a functional copy of the HBB gene, which is responsible for making the beta-globin unit of hemoglobin.