Dear members of the Haemophilia community,
At Roche, we respect and value the close relationships that exist within the haemophilia community. We also understand the important role patient organizations play within it. As such, we would like to officially update you on recent events in our HAVEN 1 study.
HAVEN 1 is the ongoing phase III study investigating the safety and efficacy of emicizumab in people 12 years of age or older with haemophilia A and factor VIII inhibitors.
As part of our standard process to receive and expedite the reporting and investigating of adverse drug events (AEs), we received two new reports of Serious Adverse Events (SAE) in a patient who was enrolled in the HAVEN 1 study. Unfortunately, we were informed that the patient passed away. We are deeply saddened by this information.
The HAVEN 1 trial is ongoing, and thus the amount of information we have and can share is limited in order to protect the integrity of an ongoing study. Below is a brief summary of the medical history.
It is our understanding that a patient experienced a serious rectal haemorrhage (first reported SAE) and received bypassing agents, including repeated doses of activated prothrombin complex (aPCC), after which the patient developed signs of Thrombotic Microangiopathy (TMA) (second SAE).
Based on the available details of the case and our initial assessment, we believe our current protocol guidance in the trial regarding the use of bypassing agents remains appropriate.
We will be evaluating the information from this particular case in the context of the other information from our on-going clinical trials.
For all of us at Roche, patient safety continues to be of paramount importance. We will continue to work closely with the IDMC, Steering Committee, and our Investigators to evaluate all safety events that have been observed and to ensure the proper measures are in place to assure continued patient safety.
Meantime, we would recommend that all questions related to the above safety events be referred to the Roche affiliate in your respective country. Questions from patients participating in an emicizumab clinical trial should be referred to the relevant study investigator.
Thank you for your ongoing leadership in supporting people with haemophilia.