Bluebird, winding down in Europe, withdraws another rare disease gene therapy – BioPharma Dive

Dive Brief:

Only a handful of gene therapies for inherited diseases are approved worldwide. Bluebird, holding two of them, has been one of the leading developers.

So Bluebird's struggles in Europe are notable for the dozens of other biotech companies advancing gene-based treatments for uncommon diseases like cerebral adrenoleukodystrophy or severe beta thalassemia.

The company's decision also reflects the differences in how therapies approved in Europe are paid for, with decisions on reimbursement left up to the governments of individual EU member states. Compared to the U.S., European countries can be more aggressive in demanding lower prices and, as many have single-payer healthcare systems, are better able to negotiate for larger discounts.

While Bluebird set a $1.8 million price for Zynteglo, the company proposed having countries reimburse for treatment over five years. Payments were linked to continued patient benefit.

Germany, however, countered with an initial price of $790,000 per patient, rising to roughly $950,000 if patients were benefiting from treatment several years later, according to an April report from STAT News.

That proved unpalatable to Bluebird, which pulled Zynteglo from Germany before later announcing a broader withdrawal from Europe.

"Bluebird's decision to focus on the U.S. market is driven by the challenges of achieving appropriate value recognition and market access for Zyntegloin Europe, which makes bringing its transformative gene therapies like Zyntegloand Skysona to patients and physicians in Europe untenable for a small innovative company at this time, said Andrew Obenshain, head of severe genetic diseases at Bluebird, in an August statement.

Along with the withdrawal of Skysona from Europe, Bluebird is also pulling back an application for approval in the U.K., according to a Thursday filing with U.S. securities regulators. Withdrawal of Zynteglo from the EU and U.K. will be complete by early 2022.

Bluebird said it will continue long-term follow-up of patients treated in clinical trials in Europe, but will not carry out further study of the treatments there.

While specific numbers aren't available, few patients ever received Zynteglo. The first patient ever treated commercially only received the therapy in February of this year, after manufacturing difficulties had delayed Bluebird's launch of the drug.

Neither Zynteglo or Skysona were expected to be widely used given the small patient populations they were approved to treat. But clinical testing had shown both to be effective therapies, meaning their withdrawal leaves patients in Europe with one less treatment option.

Bluebird recently asked the Food and Drug Administration for approval of Zynteglo in the U.S. and plans to do the same for Skysona by the end of this year.

The company is also in the midst of splitting in two, with its cancer research and programs set to be spun out into a new independent company called 2seventy bio.

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Bluebird, winding down in Europe, withdraws another rare disease gene therapy - BioPharma Dive