Earlier this month, Octapharma presented new data from its ongoing NuProtect Study, which was designed to measure, in part, the immunogenicity of their hemophilia A treatment Nuwiq®. The findings were presented at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3-6, 2016, in San Diego, CA.
The US Food and Drug Administration (FDA) first approved Nuwiq® in the fall of 2015, for the treatment of bleeding in children and adults with hemophilia A, or factor VIII (FVIII) deficiency. The product is indicated for on-demand treatment and control of bleeding episodes, routine prophylaxis to reduce the frequency of bleeding episodes and perioperative management of bleeding. Nuwiq® is the first B-domain-deleted recombinant FVIII derived from a human cell line. The therapy is cultured without using additives of human or animal origin, and is not chemically modified or fused with another protein.
Immunogenicity is the ability of a particular substance to trigger an immune response in the body of a human or animal. In hemophilia, this takes the form of an inhibitor antibody response to factor replacement therapy. An inhibitor’s severity is measured by the Bethesda inhibitor assay, a blood test that determines the amount of antibodies directed against the clotting factor. Results of the assay are given in Bethesda units (BU). Patients with an inhibitor titer below 5 BU are said to have a weak, low-titer inhibitor; those with a titer of 5 or more BU have a potent, high-titer inhibitor.
The investigators evaluated the use of Nuwiq® in previously untreated patients (PUPs) with severe hemophilia A. PUPs with severe hemophilia are often more susceptible to inhibitors during the first 50 exposure days to FVIII. The presentation made by Octapharma at ASH reported on the immunogenicity of Nuwiq® in 66 hemophilia A patients who received at least 20 days of treatment, with no previous exposure to FVIII concentrates or other blood products. The cumulative incidence of all inhibitors was 20.8%; 12.8% for high-titer inhibitors and 8.4% for low-titer inhibitors.
According to Octapharma, these data were reported as part of a pre-planned interim analysis for the NuProtect study, which intends to evaluate at least 100 PUPs, making it one of the largest studies with a single FVIII concentrate.
“Our goal was to design a rFVIII with reduced immunogenic potential. We are now very proud to present this significant clinical data with Nuwiq®, which we believe validates our approach in the most vulnerable patient population, and thank all the patients and investigators for their participation in the study,” said Olaf Walter, Board Member at Octapharma AG.
Source AFP, December 21, 2016
