Safety and efficacy of nonacog alfa for the treatment of haemophilia B in children younger than 6 years of age in a routine clinical care setting: the EUREKIX registry study

Abstract

Introduction European regulatory authorities request postmarketing safety and efficacy data for factor IX (FIX) products. Aim Collect additional clinical data from routine nonacog alfa use in children aged <6 years with haemophilia B. Methods The EUREKIX registry included retrospective and prospective data collection phases. Safety was assessed via adverse drug reactions (ADRs)/adverse events (AEs) and events of special interest (ESIs) as the primary objective; efficacy was evaluated via annualised bleeding rates (ABRs). Results The retrospective phase comprised 37 subjects. Of these, 25 had severe haemophilia B. One subject experienced 2 ADRs; another experienced 4 ESIs of hypersensitivity. Median ABR in subjects receiving a predominantly on‐demand regimen (prophylaxis <50% of time; n = 11) was 2.0; median ABR was 3.8 in those receiving predominantly prophylactic treatment (prophylaxis ≥50% of time; n = 24). Joint bleeding was infrequent (median ABR, 0.4; n = 35). The prospective phase included 26 subjects, with 17 continuing from the retrospective phase. A total of 20 subjects had severe haemophilia B. Three subjects experienced 7 treatment‐related AEs; 3 experienced 4 ESIs. Median ABR was 4.5 and 1.1 in subjects who received predominantly on‐demand (n = 5) or prophylactic treatment (n = 19), respectively; the overall median ABR for joint bleeding events was 0.0. Conclusions Overall, nonacog alfa treatment effectively controlled bleeding events, with no new safety signals identified. These data support the safety and efficacy of nonacog alfa in routine clinical settings in children aged <6 years.

Practices and challenges for hemophilia management under resource constraints in Thailand

“…As of 2023, recombinant CFCs remain more expensive than plasma-derived CFCs in Thailand (Table 5 ). Nonacog alpha, a recombinant SHL FIX concentrate which was recently approved by the Thai Food and Drug Administration for HB treatment, has a potential for the low-dose HB prophylaxis (e.g., 500 FIX IU/week) based upon the reimbursable budgets [ 7 ], despite the limited local experience. For HA patients with inhibitors, although emicizumab prophylaxis—outside NHSO and SSO coverage—could be used in Thailand, only 7% of patients were able to afford its out-of-pocket costs [ 3 , 8 ].…”