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Sanofi Presents Final Trial Data for rFVIII Candidate

July 22, 2019
Sanofi Presents Final Trial Data for rFVIII Candidate at ISTH

Sanofi Genzyme reported positive data from their recently completed phase 1/2 EXTEN-A study of their investigational factor replacement product for hemophilia A. BIVV001 is a recombinant factor VIII therapy (rFVIII) designed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic infusions. The data was included in an oral presentation at the XXVII Congress of the International Society on Thrombosis and Haemostasis, July 6-10, 2019 in Melbourne, Australia.

EXTEN-A was a multicenter trial designed to evaluate the safety, tolerability, and pharmacokinetics of BIVV001 in two different size doses in severe hemophilia A patients between ages 18-65 years. Enrolled individuals were placed in one of two groups, depending on the size dose they received.

Patients who received the larger dose of the therapy achieved a factor VIII (FVIII) half-life of 43 hours, representing a significant increase from the 13 hour half-life reached in response to treatment with an existing rFVIII product. Average FVIII activity for participants in the larger dose group was 38% at five days post-BIVV001 administration and 17% at seven days – these represent therapeutically significant increases from less than 1% FVIII levels that are normally found in individuals with severe hemophilia A. In the smaller dose group, patients achieved a FVIII half-life of 38 hours, signifying a notable improvement on the nine hour half-life achieved with an existing FVIII product. The company also reported that the therapy was generally well tolerated with no inhibitor development and no adverse events.

Overall, a single, prophylactic dose of BIVV001 resulted in sustained FVIII activity levels across trial participants who benefited from an extended half-life 3-to-4-fold higher than conventional rFVIII.

“These data reinforce the potential for BIVV001 to address an area of significant unmet need for people with hemophilia A; optimal protection from bleeds with longer prophylactic dosing intervals,” said Mouhamed Gueye, PharmD, MBA, Head of Global Medical Affairs, Rare Blood Disorders, Sanofi Genzyme. “Factor replacement therapy is a foundational component of hemophilia care and these early clinical results support the continued development of BIVV001.”

Source: Sanofi Genzyme press release dated July 7, 2019