Simoctocoga Alfa Effective Treatment for Hemophilia A


The study found that the infusion helped to prevent spontaneous bleeding in patients with hemophilia A.

Simoctocoga alfa, a factor VIII (FVIII) treatment, is an effective and safe treatment for hemophilia A, according to a study presented at the virtual 62nd American Society of Hematology Annual Meeting and Exposition.

Hemophilia A is a genetic bleeding disorder in which blood does not clot properly. Long-term prevention of bleeding is a key goal for both patients and care providers. The current gold standard of treatment is prophylaxis with factor FVIII.

During the initial NuProtect study, previously untreated patients were exposed to simoctocoga alfa for 100 days. The extension enrolled 48 patients at 15 centers in 9 countries, with dosing and treatment frequency being the main differences from the previous study, which was determined by care providers. The average age at time of enrollment was 2.8 years, with ages ranging from 1.3 to 11.9 years.

During the initial study, participants showed a showed a 17.6% cumulative incident of high titer inhibitors. The median annual spontaneous bleed rate was 0. By the end of the extension study, 85% of participants were on a twice-weekly infusion schedule. No patient developed FVIII inhibitors during the extension, according to the study. Of the patients enrolled, 34% experienced no bleeding episodes and 72% experienced no spontaneous bleeding episodes.

Simoctocoga alfa was well tolerated and effective and the mean follow-up was 20.6 months. Overall efficacy was performed by a hematologist and surgeon, both of whom gave a rating of “excellent.”


Liesner, Ri J. Neufeld, Ellis J. 2681 Long-Term Prophylaxis with Simoctocog Alfa for the Management of Haemophilia Α: Immunogenicity, Efficacy and Safety Results from the Nuprotect PUP Extension Study [Abstract] December 2020, American Society of Hematology. Accessed December 3, 2020.

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