On February 23, 2026, BioMarin announced the withdrawal from the EU market of Roctavian (valoctocogene roxaparvovec) a gene therapy for the treatment of hemophilia A (source: https://www.biomarin.com/news/company-statements/biomarin-voluntarily-withdraws-roctavian-from-the-market/. The news came after the Company was unable to find a buyer for the product, following the decision to offload Roctavian from the Company’s portfolio announced in October 2025. The European Commission had granted a conditional marketing authorization to Roctavian on 24 August 2022.
Roctavian is not the only advanced therapy medicinal product (ATMP) that was withdrawn from the EU market in recent years.
On 24 March 2022, the European Commission announced the withdrawal of the European marketing authorisation for Zynteglo (betibeglogene autotemcel), a gene therapy originally approved on 29 May 2019 for the treatment of transfusion-dependent β-thalassaemia (source: https://www.ema.europa.eu/en/medicines/human/EPAR/zynteglo ). The withdrawal had been requested by bluebird bio, the MAH, “for commercial reasons”.
Only a few months earlier, on 26 November 2021, the European Commission had announced the withdrawal of the European marketing authorisation for Skysona (elivaldogene autotemcel), another gene therapy developed by bluebird bio for the treatment of cerebral adrenoleukodystrophy (source: https://www.ema.europa.eu/en/documents/public-statement/public-statement-skysona-withdrawal-marketing-authorisation-european-union_en.pdf ); also in this case, the withdrawal had been requested by the MAH, “for commercial reasons”.
What are these events telling us? What do these cases have in common, apart from all of them being gene therapies, and what might be, instead, any relevant differences? How do the above stories differ from the fate of other gene therapies, e.g., Zolgesma (onasemnogene abeparvovec) for spinal muscular atrophy or Luxturna (voretigene neparvovec) for the treatment of vision loss due to inherited retinal dystrophy?
We would love discussing the above case studies with those who might have an interest in the matter of pricing, reimbursement and market access of advanced therapy medicinal products. Should anyone like to learn more, please contact us at the following email address: valuebox@valuevector.com.
