Nuwiq prevents and controls bleeds in patients new to FVIII therapy
Phase 3 trial finds hemophilia A treatment safe, effective in therapy-naive group
Nuwiq (simoctocog alfa) effectively prevented and controlled bleeds in previously untreated people with severe hemophilia A, according to data from a Phase 3 clinical trial.
Full trial results were reported in the study, “Simoctocog alfa (Nuwiq) in previously untreated patients with severe haemophilia A — Final efficacy and safety results from the NuProtect study,” published in the European Journal of Haematology. The work was funded by Nuwiq’s developer, Octapharma.
A widely approved treatment for hemophilia A, Nuwiq can be used both as a prophylactic or on-demand treatment to prevent or control bleeding episodes. The therapy contains a lab-made version of factor VIII (FVIII), the clotting protein that is missing or is faulty in people with hemophilia A.
Octapharma conducted the global, Phase 3 NuProtect trial (NCT01712438) to test Nuwiq in children and adults with hemophilia A who had not previously used any treatment. Its main goal was to assess these patients’ risk of developing inhibitors, which are antibodies that can bind to the delivered FVIII and reduce its efficacy. Results published in 2021 showed that the risk was low, with inhibitors detected in about one 1 of every 4 patients.
Effectiveness of hemophilia A treatment tested in 108 children and adults
Scientists at Octapharma and other institutions now are reporting secondary trial goal findings, including the therapy’s efficacy at controlling bleeds and its safety.
A total of 108 patients were treated with Nuwiq in the study. Among them, 103 received at least one prophylactic dose of the therapy, and 50 received continuous prophylaxis for at least 169 days (about six months).
Patients on continuous prophylaxis with Nuwiq had a median overall bleeding rate of 2.5 bleeds per year. The median rate for traumatic (injury-related) bleeds was 2.1 each year, while the median rate for spontaneous bleeds was zero, meaning that most patients on continuous prophylaxis did not experience spontaneous bleeds.
Similar observations were made in the group of patients given at least one prophylactic dose of the therapy in the trial.
Nuwiq also was used as an on-demand treatment to control over 800 bleeding episodes in 85 patients. Most of these episodes (65.1%) were classified as minor. The treatment’s overall efficacy at controlling bleeds was rated as excellent or good in more than 90% of these episodes.
Likewise, Nuwiq was used to control bleeding during 23 surgeries in 21 patients, involving 13 minor and 10 major procedures. The therapy’s efficacy at controlling surgical bleeds was rated as good or excellent in all but one procedure, given a moderate rating. All efficacy outcomes were rated by a surgeon and a blood disorder specialist.
Researchers concluded that Nuwiq “was effective in the prevention and treatment of [bleeding events], and as surgical prophylaxis, in this patient population,” adding that these findings “are consistent with data from studies of [Nuwiq] in previously treated adults and children.”
Safety for treatment-naive patients generally consistent with other studies
In terms of safety, 18 patients experienced fever that was considered to be a possible side effect of Nuwiq. Eight patients showed allergic reactions, with symptoms like rash. Two cases of fever and one allergic reaction were classified as serious events possibly or probably related to Nuwiq, as were the 28 cases of patients who developed inhibitors.
There also was a reported case of anemia (low red blood cell counts) and another case of hemorrhagic (bleeding-related) anemia, with the latter being considered severe. These two events were considered to be related to Nuwiq treatment by clinical investigators, but Octapharma disputed this interpretation, arguing these events are more likely to have been due to hemophilia A itself.
No side effects related to clotting and no deaths were reported during the study.
Overall, these safety findings are generally consistent with earlier research in patients previously treated with FVIII or other blood products. The one exception was fever, which was markedly more common in this study than in prior studies of previously treated patients.
“The higher percentage of patients who had [fever] that was rated as related by the sponsor is likely due to the fact that any [adverse event] occurring within 24 [hours] of administration was classified as related unless concurrent conditions existed that would have been a more likely cause of the [adverse event],” the researchers wrote.
Collectively, these findings “make [Nuwiq] an appealing option for [previously untreated patients] as a first and ongoing treatment for patients with severe [hemophilia A],” the scientists concluded.
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