ADVATE Efficacy ADVATE Efficacy

Proven efficacy. Extensive clinical data.

Backed by real-world experience spanning over 10 years1

Advate 12 Clinical Trials

Since 2000, ADVATE has been evaluated extensively in a large FVIII development programme in haemophilia A.1

12 trials
ADVATE Patient Willie

Over 10 years of experience lets me know i can believe in the product i'm using.

— Willie, Taking advate prophylaxis since 2003

Actual patient with haemophilia A. Individual results may vary.

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prophylaxis

Toward a world of zero bleeds

Phase 4 prophylaxis study:

Phase 4 Prophylaxis Study

study details

This multicentre (21 EU sites, 9 US sites), open-label, randomised, prospective, phase 4 clinical study (N=73) was designed to compare the efficacy of 2 ADVATE prophylactic treatment regimens, as well as between on-demand and prophylaxis treatments, in terms of ABR.2

  • Previously on-demand treated patients aged 7-59 years (n=66) with factor VIII levels ≤2% received 6 months of on-demand treatment and then were randomised to 12 months of every-second-day, standard weight-based prophylaxis (20-40 IU/kg) or every-third-day, PK-tailored prophylaxis (20-80 IU/kg)2
  • Both regimens intended to maintain factor VIII trough levels at or above 1%2
  • Primary endpoint: Evaluation of efficacy in terms of ABR between 2 prophylactic regimens2
  • Secondary endpoints included efficacy assessment between 2 prophylactic regimens and initial on-demand treatment; assessment of safety, immunogenicity, and HRQoL2
  • 66 subjects comprised the intention-to-treat (ITT) analysis set, and of these, 53 (30 on standard and 23 on PK-tailored prophylaxis) comprised the per-protocol (PP) analysis set2
  • 42% of PP subjects achieved zero bleeds during 1 year of prophylaxis (3 of 30 treated with standard prophylaxis and 9 of 23 PK-driven prophylaxis)3
  • 33% of ITT subjects (22 patients) achieved zero bleeds during 1 year of prophylaxis2
view abstract of published study report
keyhole

Zero bleeds is important, because even a few bleeds may lead to long-term joint damage.4,5

reduction in bleeds when switched from on‑demand to prophylaxis2

Prophylaxis Study Reduced Bleeds

Per-protocol analysis set.

low abr across bleeding of all aetiologies and types while on any prophylaxis (PK or standard) that was tested in this study2,3

Prophylaxis Study Reduced Bleeds

Results were similar whether patients used ADVATE every 2 or every 3 days2

  • Every-third-day, PK-tailored prophylaxis used ADVATE (20-80 IU/kg) every 72 hours, targeted to maintain FVIII trough levels ≥1%2
  • Every-second-day, standard weight-based prophylaxis used ADVATE (20-40 IU/kg) every 48 hours, targeted to maintain FVIII trough levels ≥1%2
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clinical practice

Efficacy that translates to the real world

Post-Authorisation Safety Surveillance (PASS) programme:

ADVATE Clinical Practice

Study Details6

ADVATE [Octocog alfa (Recombinant Coagulation Factor VIII)] Post-Authorisation Safety Surveillance (PASS) programme (N=1188) was designed to determine the safety and effectiveness of ADVATE under conditions of routine clinical practice (as opposed to the carefully controlled setting of an investigational trial)6:

view abstract of published study report BACK TO TOP

bleed resolution

Effective bleed resolution

Phase 3 pivotal study:

86% of responses to bleeding episodes were rated excellent or good7

Haemostatic efficacy for the treatment of bleeding events was assessed in PTPs ≥10 years of age with moderate to severe haemophilia A (baseline FVIII ≤2%) as part of a phase 3 study of the safety and efficacy of ADVATE.7

ADVATE Bleed Resolution

Study Details7

  • Multicentre phase 3 clinical study (N=108) of PTPs (≥150 exposure days to other FVIII concentrates) with moderate to severe haemophilia A (baseline FVIII ≤2%)7
  • Subjects self-administered ADVATE [Octocog alfa (Recombinant Coagulation Factor VIII)] both for on-demand treatment and for prophylaxis over 75 exposure days7
    • Prophylactic regimen: ≥25 IU/kg 3–4 times per week
  • Pharmacokinetics was evaluated during the double-blind, randomised, crossover portion of the study7
  • Safety and haemostatic efficacy were rated during the open-label, uncontrolled phase of the study7
View abstract of published study report
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treatment-naïve

Highly effective in PUPs

Efficacy results in treatment-naïve patients:

Previously untreated patients and minimally treated patients respond well to ADVATE [Octocog alfa (Recombinant Coagulation Factor VIII)]8

  • 90% of bleeding episodes (n=463/517) were managed with 1 or 2 infusions8
  • Of the bleeding episodes rated for efficacy (n=466), 93% were categorised as excellent or good8
  • 100% of perioperative treatments were rated excellent or good in surgeries performed during the study (n=27)8
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paediatric

efficacy profile in PTPs <6 years of age

Effective in paediatric patients:

Highly rated haemostatic efficacy and prophylaxis results9

  • Resolution of bleeding was rated excellent or good in 94% of bleeding episodes9
  • No joint bleeds were experienced by patients on prophylaxis9
  • Median (range) annual joint bleeds were 0.0 (0.0-5.8) for standard prophylaxis, 0.0 (0.0-6.1) for modified prophylaxis, and 14.2 (0.0-34.5) for on-demand treatment
ADVATE Paediatric

Study details9

  • Multicentre, open-label, prospective, clinical study of previously treated paediatric patients (N=53)
  • Patients were <6 years of age at enrolment, with severe or moderately severe haemophilia A (baseline FVIII ≤2%) and ≥50 exposure days to other FVIII concentrates
  • Subjects were assigned to 1 of 3 treatment regimens at the discretion of the investigator, and could be switched between regimens at any time
    • On-demand treatment
    • Standard prophylaxis (25–50 IU/kg 3–4 times per week)
    • Modified prophylaxis (dose and/or frequency differing from standard prophylaxis)
  • Efficacy assessments included effect of regimen on both overall and joint ABRs, and the number of infusions required to treat bleeding episodes effectively
  • Pharmacokinetics and safety were monitored over a median of 156 exposure days
    • No inhibitors were detected
    • No treatment-related serious adverse events were seen
view abstract of published study report
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surgery

excellent haemostatic efficacy

Surgery study results:

ADVATE [Octocog alfa (Recombinant Coagulation Factor VIII)] received top ratings for perioperative management†10

100%

In 61 of 61 intraoperative procedures, ADVATE received a rating of excellent or good10

100%

In 62 of 62 postoperative procedures, ADVATE received a rating of excellent or good10

91%

Hemostatic efficacy was rated excellent or good in 20 of 22 surgical drain removal procedures10

Haemostatic efficacy was rated on a standard 4-point scale. Evaluations were done by the patient for home treatment, or by the investigator for hospital or clinic treatment.

view abstract of published study report
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prophylaxis qol

ADVATE prophylaxis improves patients'
quality of life

compared to on-demand treatment

After 1 year of ADVATE prophylaxis, patients had statistically significant and clinically meaningful improvements in Physical Component Score (p=0.0002) and subcategory of Bodily Pain (p=0.0007) compared with on-demand treatment.†2

ADVATE Prophylaxis QOL

After 1 year of ADVATE prophylaxis, patients had statistically significant and clinically meaningful improvements in Physical Component Score (p=0.0002) and subcategory of Bodily Pain (p=0.0007) compared with on-demand treatment.†2

ADVATE Prophylaxis QOL

Patients (14 years and older) were asked to complete the SF-36 health survey after each treatment period (n=57). Clinically significant changes were not seen in the physical health–related subcategories of General Health and Physical Functioning and the Mental Health Component Score and subcategories of Mental Health, Role Emotional, Social Functioning, and Vitality.2
The subcategories of Physical Functioning, Role Physical, and Bodily Pain correlate highly with the physical component and contribute most to scoring of the Physical Component Summary measure.11
§Measures intensity of bodily pain and extent of interference with work due to pain.11

view abstract of published study report
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Summary of the breadth of ADVATE clinical data1

  • Phase 4 prophylaxis study (N=73)
  • PUP study (N=55)
  • Phase 2/3 pivotal trial (N=108)
  • Phase 2/3b continuation study (N=82)
  • Paediatric study (N=53)
  • Surgery study (N=59)
  • Japanese registry study (N=15)
  • Dose-response study (N=25)
  • Canadian paediatric continuation study (N=4)
  • 2 ml reconstitution bioequivalence study (N=42)
  • Bonn study (N=9)
  • High-potency study (N=23)
ADVATE Patient Matt

before i was on advate prophylaxis, i would have 2-3 bleeds a month, and now i’ve been able to achieve zero bleeds.

— Matt, Taking advate prophylaxis since 2003

Actual patient with haemophilia A. Individual results may vary.