espectively (see Table 8). The mean ABR (SD) for treatedbleeds and all bleeds were 3.1 (7.1) and 4.4 (8.7), respectively. Of the 68 children, 22 (32%) did not experience any bleedingepisodes and 29 (43%) did not experience any bleeding episodes that required treatment during the Main Phase of the trial.
Of the 13 subjects with 17 documented target joints at baseline, 10 subjects (77%) and 14 target joints (82%) did not haveany bleeds during the Main Phase of the trial.
Table 8: Efficacy in pediatric prophylaxis, medianand mean ABR by age and bleed type
Prophylaxis Regimen
Age range < 6 years** 6 to < 12 years 0 to < 12 years
# of subjects N=34 N=34 N=68
Mean treatment duration
(years)
0.46 0.51 0.48
Treated bleeds
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds
Median ABR (IQR)
Mean ABR (SD)
19 (56)
15 (44)
30
1.9 (0.0;2.1)
3.9 (9.7)
20 (59)
14 (41)
40
2.0 (0.0;3.9)
2.3 (2.9)
39 (57)
29 (43)
70
2.0 (0.0;2.8)
3.1 (7.1)
All Bleeds (treated and
untreated)
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds*
Median ABR (IQR)
Mean ABR (SD)
20 (59)
14 (41)
41
2.0 (0.0;4.0)
5.0 (11.9)
26 (77)
8 (24)
65
2.0 (1.9;6.0)
3.8 (3.6)
46 (68)
22 (32)
106
2.0 (0.0;4.2)
4.4 (8.7)
Treated spontaneous bleeds
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds
Median AsBR (IQR)
Mean AsBR (SD)
6 (18)
28 (82)
9
0.0 (0.0;0.0)
2.1 (7.3)
7 (21)
27 (79)
10
0.0 (0.0; 0.0)
0.6 (1.5)
13 (19)
55 (81)
19
0.0 (0.0; 0.0)
1.3 (5.3)
Treated traumatic bleeds
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds
Median AtBR (IQR)
Mean AtBR (SD)
15 (44)
19 (56)
20
0.0 (0.0; 2.0)
1.7 (4.0)
17 (50)
17 (50)
30
0.9 (0.0;2.0)
1.7 (2.5)
32 (47)
36 (53)
50
0.0 (0.0;2.0)
1.7 (3.3)
Treated joint bleeds
# of subjects with bleeds (%)
# of subjects without bleeds (%)
# of bleeds
Median AjBR (IQR)
Mean AjBR (SD)
7 (21)
27 (79)
10
0.0 (0.0;0.0)
1.5 (6.3)
12 (35)
22 (65)
24
0.0 (0.0;2.0)
1.4 (2.4)
19 (28)
49 (72)
34
0.0 (0.0;2.0)
1.5 (4.7)
ABR = annualized bleed rate; IQR = interquartile range, 25th percentile to 75th percentile; SD = standard deviation; AsBR = annualized spontaneous bleed
rate; AtBR = annualized traumatic bleed rate; AjBR = annualized joint bleed rate
*Reflects all bleeds reported by patients including those where no ESPEROCT was administered
**Elevated mean ABRs are due to subjects who withdrew from the study, whose bleeding rates were extrapolated to one year16 HOW SUPPLIED/STORAGE AND HANDLINGHowSupplied
• ESPEROCT is supplied in packages comprised of a single-dose vial containing nominally 500, 1000, 1500, 2000 or3000 IU of Factor VIII activity; a MixPro® pre-filled diluent syringe containing 0.9% saline solution; and a sterilevial adapter with a 25-micrometer filter, which serves as a needleless reconstitution device.
• The actual Factor VIII activity in IU is stated on each ESPEROCT carton and vial label.
Table 9: ESPEROCT Presentations
Nominal Dosage
Strength
Cap Color
Indicator
Carton NDC
Number
Components
500 IU Red NDC 0169 8500 01 • ESPEROCT in single-dose vial [NDC 0169 8501 11]
• Pre-filled syringe with 4 mL sterile saline diluent[NDC 0169 8008 98]
• Vial adapter1000 IU Green NDC 0169 8100 01 • ESPEROCT in single-dose vial [NDC 0169 8101 11]
&b |
