0)
4.0 (0.0; 8.0)
Month 1-6†: 4.1;
Month 7-12†: 1.1
2.0 (0.0; 4.9)
Month 1-6†: 2.0;
Month 7-12†: 2.0
Spontaneous Bleeds
0.5 (0.0; 2.0)
1.0 (0.0; 3.9)
2.0 (0.0; 6.5)
0.0 (0.0; 3.0)
Joint Bleeds
0.5 (0.0; 7.0)
1.8 (0.0; 3.0)
2.5 (0.0; 7.5)
1.0 (0.0; 4.0)
Subjects with Zero Bleeding Episodes‡ % (n)
37.5% (6/16§)
62.5% (10/16§)
28.6% (8/28¶)
25.8% (8/31¶)
The ABR for subjects (n=21) receiving on-demand therapy in Study 2 [median (IQR Q1; Q3)] for all bleeds: 60 (41.7; 76.3); spontaneous bleeds: 42.1 (24.3; 61.3); joint bleeds: 38.8 (24.3; 60.0).
Children 12 Years of Age and Younger
A total of 51 PTPs were treated with KOVALTRY for at least 6 months with median (range) 73 EDs (37-103) (see TABLE 13). Subjects received >95% of the prescribed number of prophylaxis infusions.
Table 13: Prophylaxis Treatment with KOVALTRY in Children 12 Years of Age or Younger – Treatment Exposure
Study 3
PTPs 0 to <6 yrs
(N=25) PTPs 6 to 12 yrs
(N=26)
* Treatment regimen at the start of the study. Study duration was six months.
Treatment regimen* during study (6 months) n (%)
2 times per week
3 times per week or every other day
9 (36%)
16 (64%)
13 (50%)
13 (50%)
Nominal prophylaxis dose per infusion, median (range)
36.4 IU/kg (21-58 IU/kg)
31.8 IU/kg (22-50 IU/kg)
In children 12 years of age and younger (n=51), the median (IQR Q1; Q3) ABR within 48 hours after prophylactic infusion was 0 (0; 4) for all bleeds, and 0 (0; 0) for spontaneous and joint bleeds. The median (IQR Q1; Q3) ABR during prophylactic treatment independent of time of infusion was 1.9 (0; 6) for all bleeds, 0 (0; 0) for spontaneous bleeds and 0 (0; 2) for joint bleeds. The mean ABR within 48 hours after prophylactic infusion was 2.04 ± 2.91. The mean ABR at any time during the prophylaxis regimen was 3.75 ± 4.98.
In both age groups (0 to <6 years and 6 to 12 years), the ABR for spontaneous bleeds and joint bleeds within 48 hours after prophylactic treatment [ABR median (IQR Q1; Q3)] was 0 (0; 0). The median (IQR Q1; Q3) annualized number of spontaneous bleeds during prophylactic treatment independent of time of infusion was 0 (0; 0). The median (IQR Q1; Q3) annualized number of joint bleeds during prophylactic treatment independent of time of infusion was 0 (0; 1.9) in 0 to <6 years age group and 0 (0; 2.1) in 6 to 12 years age group (see TABLE 14).
The majority (32/53) of bleeds that occurred within 48 hours after a previous prophylaxis infusion were trauma related. Twenty-three (45.1%) subjects reported no bleeds during the six-month prophylaxis period.
Table 14: ABR in Children 12 Years of Age or Younger
Study 3
PTPs 0 to <6 yrs
(N=25) PTPs 6 to 12 yrs
(N=26)
* Independent of time of infusion † IQR = Interquartile Range
Within 48 hrs after prophylactic treatment
During prophylactic treatment*
Within 48 hrs after prophylactic treatment
During prophylactic treatment*
All Bleeds ABR Median (IQR†Q1; Q3)
1.9 (0.0; 4.0)
2.0 (0.0; 6.0)
0.0 (0.0; 2.0)
0.9 (0.0; 5.8)
Number of Subjects with Zero Bleeding Episodes (%)
10 (40%)
13 (50%)
16 HOW SUPPLIED/STORAGE AND HANDLING
How Supplied
KOVALTRY is available as a lyophilized powder in single-use glass vials, one vial per carton. It is supplied with a sterile vial adapter with 15-microm
