Wolfgang Miesbach: What Goes Wrong When Approved Gene Therapies Get Withdrawn?
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 Gene therapy withdrawn.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38% of all approved ATMPs in the EU have been withdrawn – not because they failed, but because the commercial model collapsed.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 as marketing authorization holders — Telethon took over Strimvelis from Orchard Therapeutics in 2023, the first such transfer for an ATMP
- The AGORA Foundation enabling cross-border access to proven therapies across Europe
- Public benefit corporations like Rarity (US) advancing ADA-SCID gene therapy
- Hospital exemption pathways as a structured route for ultrarare diseases
- EU joint clinical assessments now operational from 2025 — a first step toward harmonized reimbursement
For haemophilia, this is not hypothetical – it is happening now.
The lessons from immune deficiency gene therapy are directly relevant to where we stand with hemophilia A and B gene therapies today.
Congratulations to the authors Ana Hidalgo-Simon and Claire Booth.”
Title: Advancing ATMPs for orphan diseases: redefining the roles of pharmaceutical companies and academia
Authors: Ana Hidalgo-Simon, Claire Booth
Read the Full Article on Blood ICT
Stay updated on all scientific advances with Hemostasis Today.
-
Jul 7, 2026, 06:19Simone Schoenwaelder: Women Are 25% More Likely to Have Their Stroke Misdiagnosed and Here’s Why
-
Jul 7, 2026, 06:12Dhiraj Bhatia: Extremely Happy to Share Our Review on DNA Nanotechnology in Stroke Repair
-
Jul 7, 2026, 06:04Brian A Beh: An Open Letter to Lisa Murphy Honouring the 30 Years of the Stroke Foundation’s Dedication
-
Jul 7, 2026, 05:55Alan Nurden: A Landmark Review Comparing Hemophilia and Hereditary Angioedema
-
Jul 7, 2026, 05:40Andrew Agwunobi: UConn Health is Proud to Advance Stroke Rehabilitation for Veterans
-
Jul 6, 2026, 22:54Louise St. Germain Bannon: I Wanted to Recognize the Incredible Staff Behind ISTH 2026
-
Jul 6, 2026, 21:46Armghan Ans: UPMC Washington Stroke Program Earns AHA Gold Plus Award for 16th Consecutive Year
-
Jul 6, 2026, 20:31Manik Madaan: Master Transfusion Reactions for USMLE In Less Than 60 Seconds
-
Jul 6, 2026, 19:25Ambreen Kashif: Highlighting the Impact of Temporary Donor Deferrals on Donor Retention