Nuwiq is a medication approved by the U. S. Food and Drug Administration (FDA) for the treatment and prophylaxis of bleeding episodes, and management of bleeding during and after surgery, in patients with hemophilia A. The treatment also is approved in the E.U., Canada, Australia, Latin America, and Russia. It is marketed by Octapharma.
How Nuwiq works
Hemophilia A patients lack functional clotting factor VIII, a protein needed for blood clotting. Nuwiq is a recombinant clotting factor VIII concentrate. It is administered by infusion as a prophylactic treatment or for on-demand management of bleeding episodes.
Besides Nuwiq, there are several other factor VIII concentrates on the market. A common problem with clotting factor therapy is that patients frequently develop antibodies or inhibitors against the clotting factor, making the treatment less effective.
Nuwiq is made from a human cell line and does not contain any modifications, nor is it fused to any other protein. Clotting factors are often modified to improve their stability, reducing infusion frequency. However, these modifications may trigger an immune response, in which the body develops antibodies against the delivered clotting factors.
Nuwiq also is free from any additives of human or animal origin, which can also promote antibody development. It is the only recombinant clotting factor that does not contain a B domain. The B domain is a part of the human factor VIII but is cleaved out during the activation of the protein. Thus, Nuwiq is identical to the active form of human clotting factor VIII.
Nuwiq in clinical trials
In a total of seven studies of a total 201 hemophilia A patients overall — including 59 under age 12 — who had been previously treated with clotting factor VIII, none developed inhibitors against Nuwiq.
An ongoing Phase 3 clinical trial (NCT01712438) is assessing the inhibitor development rate against Nuwiq treatment in previously untreated patients with severe hemophilia A. Participants receive Nuwiq for standard prophylaxis or on-demand treatment. The recommended dose is 30-50 IU of factor VIII per Kg body weight, but the treating physician decides on the dose depending on the patient’s situation.
The trial includes 110 patients, and interim data have already been made available from 66 patients who had at least 20 exposure days, which is when the inhibitors are expected to develop. According to these data, inhibitors were detected in 13 of the 66 patients. Eight patients developed an elevated concentration of inhibitors, and five patients developed a low inhibitor concentration. The authors concluded that Nuwiq has a convincing efficacy and tolerability in previously untreated patients.
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