Two gene therapies against hemophilia under trial

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Written By Rivera Claudia

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Berlin. Data collection for two new gene therapies (single infusions) against hemophilia began this Friday (August 30) as a comparative registration study. The G-BA announces this.

After •Valoctocogen Roxaparvovec (Roctavian® against hemophilia A from BioMarin) and etranacogene dezaparvovec (Hemgenix® against hemophilia B from CSL Behring) may only be prescribed by providers who document their treatment data in the German Hemophilia Register (DHR).

The G-BA provides detailed, product-specific information about the two registration studies on its website. The data collection should be used “for a renewed assessment of the benefits and to show whether the active ingredients have a relevant additional benefit for the patient compared to previously existing therapeutic options,” according to the autonomous body.

Valoctocogene roxaparvovec is compared with factor VIII preparations and emicizumab, etranacogene dezaparvovec with factor IX preparations. The observation is planned for five years; the manufacturers should have submitted the “final results” to the G-BA by 2029 at the latest, it continues. (cw)

link to the information page “Follow-up data collection for Valoctocogen Roxaparvovec (Roctavian®)”

link to the Etranacogene Dezaparvovec (Hemgenix) information page

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