Clinical experience with moroctocog alfa (AF-CC) in younger paediatric patients with severe haemophilia A: Two open-label studies

IntroductionThe pharmacokinetics (PK), efficacy and safety of moroctocog alfa (AF-CC) have been demonstrated in haemophilia A patients aged 6years. AimThese studies aimed to further describe moroctocog alfa (AF-CC) experience in paediatric patients (<12years) with severe haemophilia A (FVIII:C<1%). MethodsTwo prospective, open-label studies enrolled patients aged <12years: one study with 37 previously treated patients (PTPs) and another with 23 previously untreated patients (PUPs). All patients initially received 50IU/kg of moroctocog alfa (AF-CC) to evaluate either recovery alone, or with other PK parameters (6 to <12years) before continuing treatment for 100 exposure days (EDs) or 24months. ResultsAt baseline, mean (SD) recovery ranged between 1.32 +/- 0.65 (PUPs aged <2years) and 2.13 +/- 0.82 (PTPs aged 6 to <12years). The mean (+/- SD) half-life was 9.12 +/- 1.94hours in PTPs aged 6 to <12years. No new safety signals were detected in either study, 2 transient lower titre inhibitors occurred in PTPs while 8 inhibitors (3 low and 5 high titre) were detected in PUPs. Most bleeding episodes resolved with one infusion (94% [893/954]). The annualised bleeding rate (ABR) in the PTP study was 27.5 and 4.2 for patients reporting an on-demand and routine prophylaxis regimen at baseline, respectively. In the PUP study, the overall ABR was 5.9. ConclusionMoroctocog alfa (AF-CC) had expected PK findings (lower recovery in young children compared with older children) along with being safe and efficacious in a population of young severe haemophilia A patients.