The European Haemophilia Consortium’s (EHC) periodic review of novel treatments in haemophilia and other bleeding disorders is a review entirely developed by the EHC to help educate EHC National Member Organisations (NMOs) about the rapidly changing landscape of medicinal treatment in rare bleeding disorders. The EHC encourages its NMOs to use and adapt this document to their national needs but takes no responsibility for any changes.

The review covers recent major developments and is divided by different types of disorders for which there is an update to report. The information provided in this publication is compiled from multiple sources, including presentations at recent scientific meetings, websites, financial information and writing directly to pharmaceutical companies. It is then redrafted and presented in easy-to-understand language. This review is updated periodically.

The EHC is grateful for the work and support of the Novel Treatment Review Committee, which has overseen the content and production of this document. The EHC also wishes to give Miguel Crato, EHC President, special thanks for his work on these publications.


The name “European Haemophilia Consortium (EHC)” and the EHC logo are the exclusive property of the European Haemophilia Consortium.  Any use of the name or logo must receive prior approval from the EHC Steering Committee. The use of the EHC name and/or the EHC logo is only allowed when the content of the publication is reviewed and approved by the EHC. This rule entails all forms of publication, including print and digital formats. Reprinting, redistribution, or translation of any EHC publication is subject to the permission request.

Requests for review and approval should be submitted to the EHC Communications Team at [email protected] in a timely manner.

Please note:  Information and materials provided in Novel Treatment Review are intended for general information only. The EHC does not endorse or promote any specific pharmaceutical organisation, drug or other medical intervention; any reference to a product name is not an endorsement by the EHC.

We hope that the information provided herein is useful and we are available for any questions.