Rurioctocog alfa pegol (1) – Adynovi®

• Clinical trials
  • Study 261201 is an open-label, two-arm Phase II/IIIstudy comparing on-demand treatment versus prophylactic treatment with rurioctocog alfa pegol in previously treated adolescent (12 to < 18 years) and adult male patients (≥ 18 to 65 years) with severe haemophilia A.
  • The single-arm study 261204 investigated previously treated male patients with severe haemophilia A who were undergoing surgery or other invasive procedures.

Patients aged 12 years and over with haemophilia A (congenital factor VIII deficiency)

• For the treatment and prophylaxis of bleeding in patients aged 12 years and over with haemophilia A (congenital factor VIII deficiency), the additional benefit compared with the appropriate comparator therapy is not proven.
• The pharmaceutical manufacturer has not provided any relevant data for the assessment of the additional benefit of rurioctocog alfa pegol.
• The G-BA considers that, taken as a whole, the results presented are not sufficient to allow patient-relevant effects to be derived regarding the additional benefit of Rurioctocog alfa pegol compared with the appropriate comparator therapy.
• Conclusion
  • The pharmaceutical manufacturer has not provided any relevant data for the assessment of the additional benefit of Rurioctocog alfa pegol. The G-BA considers that, taken as a whole, the results presented are not sufficient to allow the derivation of patient-relevant effects regarding the additional benefit of Rurioctocog alfa pegol compared with the appropriate comparator therapy. The additional benefit compared with the appropriate comparator therapy is therefore not proven.

Courtesy translation only, please refer to the German original.