Pivotal study design in patients 12 years and older
The pivotal phase II/III, multicenter, open-label, nonrandomized study (N=137) evaluated the efficacy, PK, and safety of ADYNOVATE® twice-weekly prophylaxis vs on-demand treatment, and determined hemostatic efficacy in the treatment of bleeding episodes for 6 months.1
The primary endpoint was to compare the ABR between the prophylactic and on-demand treatment regimens.3
Prophylaxis (40-50 IU/kg, twice-weekly) vs on-demand (10-60 IU/kg) treatment1,3
ABR=annualized bleed rate.
*Treated population=subjects assigned to routine prophylaxis or on-demand treatment with ADYNOVATE and who received at least one dose of the product.
†N=120 in the prophylaxis-arm treated population; one patient received ADVATE® [Antihemophilic Factor (Recombinant)] during initial PK analysis, and was assigned to ADYNOVATE prophylaxis, but did not receive any ADYNOVATE treatment.
‡Per-protocol population=all subjects who were assigned to the prophylactic or the on-demand treatment regimen, treated with their originally assigned dose for the entire duration of study participation and who fulfilled the protocol compliance requirements.
Proven prophylaxis in children and adults (12 years and older)
In the 6-month clinical study: reduction in bleeds when switched from on-demand to prophylaxis.1
Striving toward a goal of zero bleeds with ADYNOVATE® prophylaxis
During the 6-month clinical study in children and adults (12 years and older) receiving twice-weekly prophylaxis treatment1:
ABR=annualized bleed rate; IQR=interquartile range.
Prophylaxis helped some patients stay bleed-free
During the 6-month study in children and adults (12 years and older)1:
Effective bleed control in children and adults (12 years and older)
When bleeds did occur in either study arm, the majority were controlled with a single infusion of ADYNOVATE®.1
In the clinical trial, bleeding episodes controlled:
*Excellent defined as full relief of pain and objective signs of bleeding cessation; Good defined as definite pain relief and/or improvement in signs of bleeding.1
Designed to extend FVIII circulation time
In the adolescent and adult (12 years and older) pivotal study, a PK comparison of ADYNOVATE® vs ADVATE® [Antihemophilic Factor (Recombinant)] was conducted in 26 subjects prior to initiation of prophylactic treatment with ADYNOVATE.5
The extended terminal half-life of ADYNOVATE was 1.4X–1.5X longer than that of ADVATE (based on one-stage clotting and chromogenic assay, respectively).5
PEGylation Technology* was deliberately chosen because
- It allows for half-life extension while maintaining the integrity of the ADVATE protein5,6
- It has a long history of use as an extension technology7
*Proprietary PEGylation Technology exclusively licensed from Nektar Therapeutics.