Simoctocog Alfa Effective as First Treatment for Hemophilia A

Simoctocog alfa, a fourth-generation recombinant factor VIII (FVIII) treatment, was shown to be effective for managing bleeding episodes in previously treated patients with severe hemophilia A. Researchers subsequently evaluated simoctocog alfa in treatment-naïve patients with severe hemophilia A during the NuProtect trial.

According to the study’s lead author, Mary Mathias, simoctocog alfa was effective and well-tolerated for prophylaxis, surgical prophylaxis, and the treatment of bleeding events in previously untreated patients with severe hemophilia A. Findings were presented in the European Journal of Haematology.

Simoctocog Alfa for Hemophilia A Treatment

The NuProtect study enrolled 108 previously untreated patients with hemophilia A. Of those patients, 16.2% had high-titre inhibitors. Authors noted no patients with non-null FVIII mutations developed inhibitors.

The efficacy of simoctocog alfa as prophylaxis during inhibitor-free periods was evaluated with annualized bleeding rates (ABRs), and efficacy as surgical prophylaxis and bleeding treatment were both evaluated with a 4-point scale.

A total of 103 patients received at least 1 dose of simoctocog alfa as prophylaxis, and 50 patients received continuous prophylaxis for at least 24 weeks.

Among patients on continuous prophylaxis, spontaneous bleeding events had a median and mean ABR of 0.0 and 0.5, and all bleeding events had a median and mean ABR of 2.5 and 3.6, respectively.

In 85 patients who had bleeding episodes, the efficacy of treatment was rated excellent or good for 747 (92.9%) out of 804 rated events. Authors added 92.3% of events were treated with 1 or 2 infusions.

Additionally, the efficacy of surgical prophylaxis was rated excellent or good for 18 (94.7%) out of 19 rated procedures. There were reportedly no safety concerns or thromboembolic events.

Mathias and colleagues suggested their results “make simoctocog alfa an appealing option for [previously untreated patients] as a first and ongoing treatment for patients with severe hemophilia A.”

Related: Fitusiran Prophylaxis in Hemophilia With or Without Inhibitors