Efficacy profile

Kovaltry demonstrated efficacy data in ABR for children with at least 2x/week prophylaxis infusions¹*

The median ABR in children ≤12 years old was 0 (Range: 0.0–12.0) for spontaneous, 0 (0.0–17.7) for traumatic and 0 (Range: 0.0–15.8) for joint bleeds^1,5*^†‡

ABR in children in LEOPOLD Kids (Part A)^1,5*†‡

Adapted from Kovaltry Product Monograph and Ljung R, et al.

Dosing frequency
at end of study
(investigator assigned)⁵

41 %

at 2x weekly

43 %

at 3x weekly

16 %

at every other day

Kovaltry demonstrated efficacy data in ABR for adults
and adolescents^1§

ABR in adolescent and adult patients in LEOPOLD I^1‡§¶

Adapted from Kovaltry Product Monograph.

Dosing frequency
(investigator assigned)¹

29 %

at 2x weekly

71 %

at 3x weekly

Kovaltry has demonstrated efficacy data with routine prophylaxis in all of the following age groups:¹*^§

LEOPOLD Kids

PTPs
0 to <6 years

2.03
Median overall ABR
(Range=0.0, 18.1) (n=25)

PTPs
6 to <12 years

0.93
Median overall ABR
(Range=0.0, 17.7) (n=26)

LEOPOLD I

Adolescents
(>12 years of age) and adults

1.03^||
Median overall ABR
(Range=0.0, 26.1) (n=62)

For the LEOPOLD I and LEOPOLD Kids studies, dosing and regimens were determined by physicians.¹

Control of bleeding episodes¹

Adolescents and adults

Total bleeding episodes treated with Kovaltry: 1887 in 108 subjects
Median consumption of Kovaltry for the treatment of breakthrough bleeds in the main studies:
- LEOPOLD I: 28.6 IU/kg/injection (range 13-54 IU/kg)
- LEOPOLD II: 28 IU/kg (range 11-49 IU/kg)
Majority of bleeding episodes were resolved with one or two infusions of Kovaltry
- 87.6% in LEOPOLD I
- 96.2% and 95.3% in prophylaxis and on-demand in LEOPOLD II

Previously treated children ≤12

Total bleeding episodes treated with Kovaltry in LEOPOLD Kids Part A main study: 97 in 28 previously treated subjects
Median consumption of Kovaltry for the treatment of breakthrough bleeds during the 6-month treatment period:
- 36.94 IU/kg/injection (range 20.8–71.6 IU/kg)

Majority of bleeds were resolved with one to two infusions of Kovaltry (89.7%)
- 92.4% in patients 0 to 6 years of age
- 86.7% in patients 6 to 12 years of age

Previously untreated and minimally treated children ≤12

Total bleeding episodes treated with Kovaltry in the LEOPOLD Kids Part B main study: 105 of the 184 bleeding episodes in 37 previously untreated and minimally treated subjects
Majority of bleeds were successfully treated with one to two infusions of Kovaltry (78.1%)

Perioperative management¹

Total major surgeries performed: 11 in 9 previously treated subjects (adults and children)
Initial Kovaltry doses administered:
- 3000–5000 IU (nominal dose) in the adolescent and adult subjects
- 2500 IU (108.7 IU/kg) was the total initial dose in a single subject ≤12 years of age who underwent a major surgery
Treatment with Kovaltry provided “good” or “excellent” hemostatic control, as assessed by surgeons

* LEOPOLD Kids (Part A): A two-part, Phase III, multicentre, open-label trial examining the safety, efficacy and pharmacokinetics of Kovaltry in previously treated patients, pediatrics <12 years, with severe hemophilia A (<1%). Patients were given prophylaxis treatment of 25–50 IU/kg at least 2x/week for treatment of breakthrough bleeds and prevention bleeds during surgical procedures.

† Dose/infusion (range) for the control and prevention of bleeding episodes: 38.7 IU/kg (20.8–71.6 IU/kg) for PTPs 0 to <6 years, 32.4 IU/kg (21.7–50.0 IU/kg) for PTPs 6–12 years and 36.9 IU/kg (20.8–71.6 IU/kg) to total PTPs 0–12 years.

‡ Intent-to-treat (ITT) population.

§ LEOPOLD I: A four-part, multicentre, open-label trial examining the safety, efficacy and pharmacokinetics of Kovaltry in previously treated patients, adults and adolescents, with severe hemophilia A (<1%). Patients in Part B were given prophylaxis treatment of 20–50 IU/kg 2–3x/week for 12 months, with two different potency assignments (CS/EP and CS/ADJ for 6 months each).^1,3

¶ Median dose/infusion (range) for the control and prevention of bleeding episodes: 31.6 IU/kg (14–67 IU/kg).

|| Data from first year of LEOPOLD I trial.