Previously untreated patients (PUPs)

Patients with haemophilia A require life-long treatment to prevent or control bleeding. During the initial period of treatment with a factor VIII (FVIII) concentrate, PUPs with severe haemophilia A are at high risk of developing FVIII-neutralising antibodies (inhibitors).1

Inhibitor development occurs in up to 40% of PUPs when treated with FVIII.2 Inhibitors interfere with the haemostatic function of FVIII treatment1 and impede future treatment with a FVIII concentrate.3 Nuwiq® (simoctocog alfa) has been proven to be effective at preventing bleeds in PUPs with haemophilia A and is associated with a low rate of inhibitor development.1

simoctocog-img

The NuProtect study

is the largest prospective study of a single recombinant Factor VIII product in PUPs 1,4

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Prospective, multicentre,
multinational, open-label,
non-controlled, phase III study

Investigated the immunogenicity,
efficacy and safety of Nuwiq® in
PUPs with severe hemophilia A

Duration: 100 exposure days
or maximum 5 years

epitope-image-0

Prospective, multicentre,
multinational, open-label,
non-controlled, phase III study

Investigated the immunogenicity,
efficacy and safety of Nuwiq® in
PUPs with severe hemophilia A

epitope-image-1
epitope-image-2

Duration: 100 exposure days
or maximum 5 years

110 PUPs enrolled

38Centers

17Countries

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110 PUPs enrolled

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epitope-img-1

38Centers

17Countries

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In the NuProtect study

Nuwiq® was associated with a low rate of inhibitor development in PUPs

of previously untreated patients treated with Nuwiq® developed high-titre inhibitors1

Cumulative inhibitor incidence

Probability of inhibitor development (%)

0 5 10 15 20 25 30 0 20 40 60 80 100 120

All inhibitors

High-titre*

Low-titre*

Exposure days
*High-titre: ≥5 BU/mL; low-titre ≥0.6 to <5 BU/mL.
F8 mutation type is a strong predictor of inhibitor development risk5

No patients with non-null F8 gene mutations developed inhibitors to Nuwiq®1

Inhibitor incidence by F8 mutation*
Cumulative inhibitor incidence (%)
60
50
40
30
20
10
0
no=
31
90
(Nuwiq®)
0
12
31
101
SIPPET
(pdFVIII)
0
16
47
96
SIPPET
(hamster cell line-
derived rFVIII)
43
22
  • F8 null mutations
  • F8 non-null mutations
*Intron 22 inversions, intron 1 inversions, nonsense mutations, splice site mutations, small duplications, small deletions (excluding in-frame and within a poly-A run) and large deletions were classified as null mutations. All other mutations were classified as non-null mutations.

Nuwiq® has been proven effective

for prophylaxis in PUPs with haemophilia A 

Efficacy of Nuwiq® in the NuProtect study

simoctocog-img

Median annualised bleeding rate for
spontaneous bleeds in PUPs on
continuous Nuwiq® prophylaxis for ≥6
months4

NuProtect Study results
Nuwiq® was developed with the aim to reduce the rate of inhibitors in PUPs 6-7

Nuwiq® is the only recombinant FVIII
produced in a human cell line without
chemical modification or protein fusion6-8

The Protect-NOW study is collecting real-world
data on how Nuwiq® is used to treat
PUPs and minimally treated patients in
everyday clinical practice

F8 gene mutation analysis service

Knowing the underlying F8
gene mutation of a haemophilia
A patient (and whether null or
non-null) will help ...

8CHECK

  • To predict whether a patient is at high or low risk
    of developing inhibitors

  • Consider the most suitable treatment for each
    individual patient

  • Free service offered by Octapharma

  • For all patients who have not had previous F8 gene
    variant analysis

For non-US-based health care professionals, send an email to eightcheck@Octapharma.com for detailed information
For US-based health care professionals,
find further information here
Disclaimer: Octapharma will not receive any sample or patient data.

References

  • 1

    Liesner RJ et al. Thromb Haemost 2021; 121:1400-8;​

  • 2

    Carcao M et al. Haemophilia 2019; 25:676-843;​

  • 3

    Carcao M et al. Exp Rev Hematol 2021;14:143-8;​

  • 4

    Mathias M et al. Haemophilia 2023; 29:1005-12; ​

  • 5

    Gouw SC et al. Thromb Hemost 2012; 119:2922-34;​

  • 6

    Kessler CM et al. Haemophilia 2015; 21 (Suppl. 1):1-12;​

  • 7

    Valentino LA et al. Haemophilia 2014; 20 (Suppl. 1):1-9;​

  • 8

    European Medicines Agency. Nuwiq Summary of Product Characteristics. Updated March 2023;​

Drug safety related information should be reported to at1safetyinformation@octapharma.com If you are a patient, please contact your physician in case of adverse reactions. Please consider the pharmacovigilance data privacy statement if you are reporting drug safety relevant events.

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