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FAQs

This website is intended to provide patients and caregivers with updated balanced clinical and scientific information regarding emicizumab. Our aim is to share new data and findings from select ongoing studies and real world use with emicizumab to provide information on the long-term safety and effectiveness of emicizumab. This information is provided only for educational purposes and is intended to support informed and meaningful treatment discussions with your healthcare providers.

You may read our letter to the hemophilia community from September 2020 about this website for additional details.

This website includes links to select published materials with information on emicizumab, including safety and effectiveness data, and additional topics that may be of interest including quality of life data, real world experience, and management topics. These materials may include scientific meeting posters and presentations and peer-reviewed publications. Alongside some of the published materials on this website are patient-friendly summaries on topics of interest for emicizumab, which have been developed by Genentech. Links to additional patient resources are also available on this website.

The current plan is for the website to be updated as key data on emicizumab is made available following journal publications or following data presentations at scientific meetings.

The PubMed links to journal publications will direct you to the National Library of Medicine database and will usually display the abstract for the specific publication. There are links to directly access the full publication on this page, however, some of these links require a subscription. If you have difficulty accessing a specific journal publication on this website, and would like to receive the full publication, you may contact our Medical Information department: (800) 821-8590, Monday-Friday, 5:00am - 5:00pm PST.

Updates on the number of fatalities in people treated with emicizumab, whether known to be related to emicizumab or not, are and will continue to be included in public safety registries and databases, such as FAERS (FDA Adverse Event Reporting System). We report to the FDA and other health authorities as required and adhere to processes to ensure ongoing compliance with regulatory requirements.

There have been no changes to the overall safety profile of emicizumab. In regards to providing case details or causality, we are not able to provide extensive information related to serious adverse events or fatalities reported following FDA approval because the level of detail available and Roche/Genentech's ability to confirm individual details is variable. We are also careful not to disclose specific details about an adverse event that could jeopardize the privacy of either the patient or their family, or breach patient confidentiality.

We have worked with experts in the field to develop a series of published articles reviewing mortality in hemophilia A and using this to establish a framework to further assess the reported fatalities in people receiving emicizumab.  These articles were first published online in the Journal of Thrombosis and Hemostasis in December 2020 and can be found on this website under the 'Additional Topics - Hemophilia A and Mortality' section under Emicizumab Data by Topics.

Patient safety is of the highest importance to us. We report to the FDA and other health authorities as required and adhere to processes to ensure ongoing compliance with regulatory requirements. Information on any safety events that impact the overall safety profile of emicizumab will be shared as quickly as possible with regulatory bodies and with physicians. Updates on the number of severe adverse events, including fatalities, in people treated with emicizumab, whether known to be related to emicizumab or not, will be shared in safety registries and databases, as well as through peer-reviewed publications and conference presentations that are planned across 2020 and beyond.

As of February 2021, more than 10,000 patients worldwide have been treated with emicizumab.  The overall benefit-risk for emicizumab remains favorable.

The last updates, published with a cutoff date of June 30, 2020, can be found on the “Safety” page of this website. These have been archived and will not be further updated. Moving forward, we will share data and findings on the safety and effectiveness of emicizumab from journal publications and presentations to the scientific community.

The quarterly updates were first established after the initial approval of emicizumab by the FDA in November 2017. The transition from clinical development to approved access for patients in many countries has meant a move from single patient reports to large databases of information on the use of emicizumab. Having a much broader availability of information from databases and individual experiences, means we can see early signals, identify trends and improve our overall understanding of the safety and efficacy profile of emicizumab. During this transition, the source of safety information has also expanded from controlled clinical trial reporting to ongoing voluntary adverse event (AE) reporting by healthcare professionals, patients and their caregivers, changing the level of detail of information about individual safety cases that we receive. In light of this transition, and so that we continue to ensure transparency, and aim to meet the community’s expectations and needs for information on hemophilia treatments, we have evolved our communication of post-approval information on emicizumab.

We are not able to contact you when this website is updated. However, the current plan is for this website to be updated as key data on emicizumab is made available following journal publications or following data presentations at scientific meetings.

The Prescribing Information along with the Medication Guide is the primary source of information on emicizumab, including the known and potential risks of emicizumab for hemophilia A with or without FVIII inhibitors. Your healthcare provider should be the primary source of information about your medical condition and the safe and effective use of any medicine, including emicizumab. You may contact our Medicine Information department with questions specific to this site: (800) 821-8590, Monday-Friday, 5:00am - 5:00pm PST.