Industry News


The Connecticut Hemophilia Society (CHS), the Hemophilia Alliance of Maine (HAM), and the New England Hemophilia Association (NEHA), work to ensure that people affected by bleeding disorders and their families living in New England remain informed about important medical information that affects the community.

On Sept. 7, 2017, ALNYLAM PHARMACEUTICALS  announced that it recently became aware of a fatal serious adverse event (SAE) that occurred in a patient with hemophilia A who was receiving fitusiran in the Phase 2 OLE study (clinical drug trial). As a result, Alnylam suspended dosing in all ongoing fitusiran studies pending further review of the safety event, and development of a risk mitigation strategy.  You can read a full press release HERE. Additionally, through conversations with Alynylam, we’ve been made aware that the SAE was an embolism (blood clot) in the brain. It is not yet known if this event is directly related to the patient’s treatment with fitusiran.

It is important to note that this product is not a ‘gene therapy’ medication, but rather modifies the expression of people’s healthy antithrombin, which causes thrombin to be increased by a subcutaneous (subQ) injection.

Times like these call our attention to the need and importance for community wide advocacy. We must be strong advocates for ourselves, and our communities.  We rely heavily on our therapy partners for future medical advancements and commend Alnylam on their transparency during this process. We take this moment to call upon our care providers and ask them to be diligent in their understanding and administration of drug trials as we all continue together towards a cure.  

If you have questions about this situation, please contact your Hemophilia Treatment Center (HTC), or your primary health care provider. Alnylam also has a hotline for patients to ask questions at: 1-866-330-0326. CHS, HAM, and NEHA will continue to monitor this case and will provide updates as they become available.  

Read the National Hemophilia Foundation’s (NHF) statement HERE.