On September 24, 2014, the medical journal, Blood, came out with a study called, Recombinant Factor VIII Products and Inhibitor Development in Previously Untreated Boys with Severe Hemophilia A. This study suggests that inhibitor incidence is higher in previously untreated patients (PUPs) with severe hemophilia A that are using Kogenate/Helixate.
The Food and Drug Administration (FDA) and European Medicines Agency (EMA) have confirmed they will be reexamining the data from this study, and will release results about their findings in early 2015.
The World Hemophilia Federation (WHF) issued a statement on October 6, and another one on November 18 expressing concerns about this study. HFA also expresses are concerns about his study, and the two others that reported similar data in 2013. HFA will continue to closely monitor this situation and continue to provide the bleeding disorders community with updates as they become available.
World Hemophilia Federation Statement, November 18, 2014
On October 3, 2014, the World Federation of Hemophilia (WFH) issued a communique regarding a study published by a group in France that demonstrated a higher than expected incidence of inhibitor development in previously untreated patients (PUPs) with severe hemophilia A treated with Kogenate FS/Bayer/Helixate NexGen compared to other recombinant factor VIII (rFVIII) products. Since then, a study published by a group from the UK has reported similar findings.
These results follow on from the unexpected results in the RODIN study, published in January 2013. That study was reviewed by regulators and in December 2013 the European Medicines Agency’s (EMA) Committee on Human Medicinal Products (CHMP) endorsed recommendations which concluded that the benefits of Kogenate FS/Bayer/Helixate NexGen continue to outweigh their risks in PUPs with hemophilia A. The EMA stated that the product information for this product should be amended to reflect the results of the RODIN study and clarify that there is no different risk between products.
Inhibitor development is caused by many risk factors, which makes it difficult to draw conclusions in a small patient population. At the moment, no firm conclusion can be made.
The WFH has requested that the US Food and Drug Administration (FDA) and the European Medicine Agency (EMA) examine all the relevant data and come to a conclusion as soon as possible. Both the FDA and the EMA have confirmed that they will be reexamining the data but they will not have the results ready before early 2015. It is the view of the WFH that all of the available data should be pooled in order to give a clearer answer about the relative risk for individual products.
Based on the currently available published data, it remains the position of the WFH that it may be prudent to consider not using Kogenate FS/Bayer/Helixate NexGen for newly diagnosed PUPs with severe hemophilia A where other safe clotting factor concentrates are available. There is no known increased risk for any other patients using these products.
The WFH will closely monitor this situation and will communicate again when further relevant information is available.