Rare Daily Staff
As Bluebird Bio winds down its operations in Europe, it is withdrawing its two approved gene therapies in the region, Skysona and Zynteglo, according to a statement filed with the U.S. Securities and Exchange Commission.
Skysona was approved by the European Commission in July for the treatment of cerebral adrenoleukodystrophy, a rare genetic neurological disorder. Zynteglo received conditional European Union marketing approval in May 2019 for the treatment of severe beta thalassemia, but manufacturing issues hampered its rollout until the beginning of 2021.
In its second quarter earnings report at the end of July 2021, Bluebird said it planned to close operations in Europe, citing difficulty in striking agreements with European governments on reimbursement for its gene therapies. Bluebird had already withdrawn Zynteglo from Germany because regulators there would not cover its $1.8 million cost.
“Bluebird’s decision to focus on the U.S. market is driven by the challenges of achieving appropriate value recognition and market access for Zynteglo in Europe, which makes bringing its transformative gene therapies like Zynteglo and Skysona to patients and physicians in Europe untenable for a small innovative company at this time,” said Andrew Obenshain, president of severe genetic diseases at Bluebird Bio, in the July statement. “While European regulators have been innovative partners in supporting accelerated regulatory paths for these therapies, European payers have not yet evolved their approach to gene therapy in a way that can recognize the innovation and the expected life-long benefit of these products. We are committed to and hope to find a potential partner who can help us carry forward our therapies in Europe.”
The company anticipates withdrawing marketing authorizations for Zynteglo from both the European Union and the United Kingdom by early 2022 but expects to continue activities for the long-term follow-up of patients previously enrolled within the European Union clinical trial programs as planned. However, it does not intend to initiate any new clinical trials in Europe for the beta-thalassemia, cerebral adrenoleukodystrophy or sickle cell disease programs.
Photo: Andrew Obenshain, president of severe genetic diseases at Bluebird Bio