BioMarin follows up on previously-reported  serious adverse event in its phase I/II gene therapy clinical trial for haemophilia A

May 5, 2022 – This is an update to the EHC’s February 4, 2022 statement regarding ‘BioMarin reporting a serious adverse event for its phase I/II gene therapy trial with valoctocogene roxaparvovec for haemophilia. Valoctocogene roxaparvovec is an experimental gene therapy (adeno-associated virus [AAV] delivering the B-domain deleted factor VIII gene) under development by BioMarin [...]

Pfizer/Sangamo Therapeutics report severe adverse event (SAE) from phase 3 AFFINE haemophilia A gene therapy study

May 5, 2022 – Pfizer/Sangamo have reported a severe adverse event (SAE) learning of a below-the-knee deep vein thrombosis (DVT) in one participant of its giroctocogene fitelparvovec clinical trial, who had elevated Factor VIII levels. A serious adverse event (SAE) is the term used to describe the occurrence of a serious health issue in a [...]

Meet Zita Gacser, EHC Think Tank Officer

We are delighted to introduce you to Zita Gacser, EHC Think Tank Officer. Zita joins Naja Skouw-Rasmussen in the Think Tank team and will be responsible for communications activities related to the Think Tank, supporting the development of the EHCucate app and supporting hybrid events.  Zita, can you tell us about yourself? I originally come [...]

Announcing: EHC Emergency Displacement Fund (EDF)

With enduring shock and sadness, we think of our community members and others affected by the current challenging times, and are working tirelessly to provide support where possible. The EHC have been working with our NMOs in the neighbouring countries and region to identify their needs, understand the key areas of support we may provide, […]

EHC Women’s Committee marks International Women’s Day

To mark the 2022 International Women's Day, held on 8 March, the EHC Women and Bleeding Disorders Committee wishes to share a pleasant surprise! The Committee has prepared an 'empty' copy of its 'diagnosis-quiz flyer (see below) so that EHC members can fill it in their local language and distribute it amongst their membership. This flyer can [...]

EHC VWD Working Group holds a strategic retreat

It was amongst much excitement and trepidation that the EHC von Willebrand Disease (VWD) Working Group (WG) finally met face-to-face in Dublin, Ireland, on Friday 28 January 2022. The EHC VWD Working Group was established in February 2020 to lead the work of the EHC VWD Platform for Europe. Still, due to the circumstances we [...]

Meet Daria Mironova, EHC Communications and Public Policy Officer

We are delighted to introduce you to our latest EHC addition, Daria Mironova. Daria joined us in December as Communications and Public Policy Officer. For this edition of our NMO Newsletter, we wish to tell you more about Daria and her role within the EHC.  Daria, can you tell us about yourself?  Yes, of course. [...]

BioMarin reports a serious adverse event for its phase I/II gene therapy trial for haemophilia A

February 4, 2022 – BioMarin’s gene therapy (valoctocogene roxaparvovec) for haemophilia A is currently in development and not yet licensed by regulators. BioMarin has completed dosing 134 patients in its phase 3 clinical study while it also continues to follow up, as recommended by regulators, on 15 patients from its ongoing phase 1/2 clinical study, […]

FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold

November 5, 2021 – On Wednesday, November 3, the U.S. Food and Drug Administration (FDA) placed the Pfizer/Sangamo haemophilia A gene therapy programme, including the pivotal phase 3 AFFINE study(NCT04370054), with giroctocogene fitelparvovec (SB-525 or PF-07055480), on clinical hold until review of a proposed protocol amendment. (Sangamo news release). This decision came subsequent to Pfizer [...]

EHCucate goes live

The EHC is delighted to announce its new educational app EHCucate. Information on rare bleeding disorders and novel therapies is abundant. It comes in various forms, making it even more complicated when searching for answers that can be complex and confusing! This is why the EHC has processed years of research and publications from reliable [...]