EMA's PRAC Wants More Study of Kogenate/Helixate Risks

Megan Brooks


November 07, 2014

In an about-face, the European Medicines Agency's (EMA's) Pharmacovigilance Risk Assessment Committee (PRAC) has recommended further assessment of the risk for factor VIII inhibitor development with the full-length second-generation factor VIII products Kogenate Bayer and Helixate NexGen, both marketed by Bayer Pharma AG.

The recommendation is based on a review of new evidence from two recently published studies, the committee said in a statement released today.

Both studies suggest that previously untreated patients with hemophilia may be at greater risk of developing inhibitors if treated with these products than if treated with other products that contain factor VIII, the committee said.

Both studies were published in Blood: one on September 24 and the other on October 22.

The development of inhibitors is a known risk with products that contain factor VIII.

In the Research of Determinants of Inhibitor Development (RODIN) study, published in the New England Journal of Medicine, previously untreated children with hemophilia A given second-generation products such as Kogenate and Helixate were more likely to develop factor VIII inhibitors than those given a third-generation recombinant product.

The RODIN study triggered an initial PRAC review.

However, the committee concluded in early December 2013 that the available data on Kogenate Bayer and Helixate NexGen did not confirm a higher risk of developing factor VIII inhibitors against these medicines when compared with other factor VIII products.

Later that same month, the EMA's Committee on Human Medicinal Products (CHMP) agreed with PRAC that the benefits of the products continue to outweigh the risks in previously untreated patients with hemophilia A, but that the product information for these medicines should be amended to include the RODIN study data.

However, in light of the two new studies just published, the PRAC today recommended a "further in-depth examination of the available evidence," the committee said.

The US Food and Drug Administration approved Kogenate/Helixate in June 2000. On the basis of the two new studies in Blood, the World Federation of Hemophilia requested in October that the administration "examine all the relevant data in an expedited manner."


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