Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn about a recent article by Ana Hidalgo-Simon and Claire Booth, published in Blood ICT:
”Gene therapy approved. Gene therapy withdrawn. What went wrong?
38% of all approved ATMPs in the EU have been withdrawn – not because they failed, but because the commercial model collapsed.
Zynteglo for β-thalassemia had orphan designation, PRIME status, conditional approval, accelerated assessment – and was still pulled from the market in 2022 when reimbursement negotiations failed.
Beqvez for haemophilia B: same story.
Why?
Most gene therapies are born in academia – outstanding science, but commercial planning is not the focus
Health systems run on chronic care budgets – a large one-off payment for a cure simply doesn’t fit
Post-approval requirements aren’t scaled for ultrarare diseases, making the economics brutal even after approval
What the authors propose is worth taking seriously:
Charitable foundations