Lonoctocog alfa (1) – Afstyla®

• Clinical trials
  • The registration trials CSL627_1001 (n = 173; age ≥ 12 to ≤ 65 years) and CSL627_3002 (n = 83; age < 12 years) are two open-label, multicentre, single-arm studies investigating the efficacy and safety of lonoctocog alfa in previously treated male patients with severe haemophilia A across different age groups.

Treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)

• For the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency), the additional benefit compared with the appropriate comparator therapy is not proven.
• The additional benefit compared with the appropriate comparator therapy is therefore not proven.
• In the dossier submitted for the assessment of the additional benefit of Lonoctocog alfa, the pharmaceutical manufacturer did not provide any direct comparative studies against the appropriate comparator therapy.
• An indirect comparison was not sought.
• The two single-arm studies form the basis of the marketing authorisation; however, they do not constitute a comparison with an appropriate comparator therapy and cannot be taken into account by the G-BA.
• The manufacturer did not intend to carry out an indirect comparison with the appropriate comparator therapy.
• The pharmaceutical manufacturer has not provided any relevant data for the assessment of the additional benefit of Lonoctocog alfa.
• The G-BA considers the results presented, taken as a whole, to be unsuitable for deriving patient-relevant effects regarding the additional benefit of Lonoctocog alfa compared with the appropriate comparator therapy.

Courtesy translation only, please refer to the German original.