Nuwiq, a fourth-generation genetically engineered version of blood-clotting factor VIII, is a safe and effective way of controlling bleeding in people with severe hemophilia A during and after surgery, a study reports.
The research, “Efficacy and safety of Nuwiq® (human-cl rhFVIII) in patients with severe haemophilia A undergoing surgical procedures,” was published in the journal Haemophilia.
Doctors often prescribe factor VIII to hemophilia A patients to prevent bleeding in case of an injury. They also give it to patients who will have surgery to prevent bleeding complications during and after an operation. It restores FVIII in the blood to normal levels until wound healing is complete.
A number of studies have demonstrated the effectiveness of genetically engineered, or recombinant, FVIII concentrates. But surgeries require intensive FVIII treatment, which raises the risk of patients developing an immune response that prevents the factor from clotting blood.
Octapharma’s Nuwiq is a human cell-line-produced recombinant FVIII processed without chemical modifications to lower the risk of an immune response. Clinical trials have shown it to be effective at both preventing and stopping bleeding in children and adults.
Researchers wanted to determine how safe and effective it was as a preventive treatment for surgery. They looked at data from seven studies involving 36 treated patients, ages 3 to 55, with severe hemophilia A. The 36 had a total of 52 operations — 25 major and 27 minor.
Nuwiq stopped bleeding in 98 percent of the surgeries — 51 of the 52. Doctors rated its effectiveness “excellent” or “good” in all except one major surgery, where it was judged “moderate.”
One to 19 Nuwiq infusions were needed for minor surgeries, and three to 76 for major ones, the results showed. As expected, higher doses were needed for major surgeries than minor ones.
None of the patients experienced serious treatment-related adverse effects, and none developed FVIII immune responses.
“These results show that Nuwiq® successfully maintained haemostasis [an ability to clot] and was well-tolerated during the perioperative and postoperative period in patients with severe haemophilia A,” the researchers concluded. The perioperative period is just before and during surgery.
Three of the study’s team of 10 researchers, based in the U.S. and across Europe, are Octapharma employees.