NUWIQ – Safety & Efficacy

POWERFUL BLEEDING CONTROL
Prophylaxis With NUWIQ Markedly Reduced Bleeding Frequency1,2

In Clinical Trials of Standard Prophylaxis With NUWIQ in Adult (N=32) and Pediatric (N=59) Patients Treated for ≥6 Months1*

ZERO

Median ABR
Spontaneous Bleeds
Adults and Children

In Adults
Median ABR for All Bleeds is 0.9

In Children
Median ABR for All Bleeds is 1.9

ABR = annual bleeding rate

*NUWIQ prophylaxis regimen: every other day or 3 times per week for ≥ 6 months.

All Patients Experienced Fewer Bleeds with NUWIQ Prophylaxis vs On-Demand Treatment With Another FVIII Product1,2†

Patients were switched from on-demand therapy (pre-study) with another FVIII product to prophylaxis with NUWIQ.

Based on negative binomial model.

EFFECTIVE BLEEDING RESOLUTION
On-Demand Treatment Readily Controlled Bleeding1

In Adult Patients Receiving On-Demand Treatment with NUWIQ3,4

N=986 bleeds evaluated

Bleeds Resolved
With 1 Infusion

Bleeds Resolved
With 1 or 2 Infusions

94% of on-demand responses to NUWIQ rated as “excellent” or “good”1

Control and Prevention of Bleeding During and After Surgery1

Thirty three surgical procedures (N=19)

In 100% of minor surgeries hemostasis rated as "excellent"

In 92% of major surgeries hemostasis rated as "excellent" or "good"

NUWIQ Safety in PTPs1

  • NUWIQ was shown to be safe in clinical studies that included 135 previously treated patients (PTPs)
  • FVIII levels can be safely and accurately measured with both the chromogenic and one-stage assays
    • No serious or severe reactions to NUWIQ in clinical studies, and no deaths reported

Inhibitor Risk in Previously Untreated Patients (PUPs)

Type of FVIII Molecule Can Affect Inhibitor Potential

In the SIPPET Study, rFVIII Products From Hamster Cells (CHO or BHK) Had a Higher Incidence of Inhibitor Development Than pdFVIII Products5

  • The SIPPET study compared the rates of inhibitors in PUPs who were treated with either plasma-derived FVIII (pdFVIII) or recombinant FVIII (rFVIII) made from hamster cells
  • Results from the SIPPET study showed:
    • PUPs treated with rFVIII from hamster cells had a 28.4% incidence of high-titer inhibitors
    • PUPs treated with pdFVIII had a 18.6% incidence of high-titer inhibitors

SIPPET: Cumulative Incidence of High-Titer Inhibitors (N=251)

Differences in high-titer inhibitor rates between pdFVIII and rFVIII were not found to be statistically significant. SIPPET authors suggested this may have been due to the small sample size of the study.

The SIPPET Study compared the incidence of inhibitors in PUPs with severe Hemophilia A treated with either pdFVIII or rFVIII derived from hamster cells (Chinese Hamster Ovary [CHO] cells or Baby Hamster Kidney [BHK] cells). Patients were followed for 50 consecutive exposure days (EDs) or 3 years or until inhibitor development was confirmed.

SIPPET = Survey of Inhibitors in Plasma-Product Exposed Toddlers; CI = confidence interval.

NuProtect Logo Final Results Confirm Low Rate of Inhibitors With NUWIQ in PUPs

NuProtect is the largest prospective study with a single FVIII product, evaluating 105 PUPs treated With NUWIQ

  • The safety of NUWIQ in PUPs with severe Hemophilia A was studied in the NuProtect trial
  • In the final analysis, data from 105 PUPs treated with NUWIQ were analyzed for inhibitor development, with 95 patients reaching ≥100 exposure days (EDs) or inhibitor development
  • Final results showed a 17.6% incidence of high-titer inhibitors

NuProtect: Cumulative Incidence of High-Titer Inhibitors (N-105)§

Final results of the NuProtect study showed a cumulative incidence of high-titer inhibitors with NUWIQ of 17.6%, which is similar to inhibitor development rates of pdFVIII seen in the SIPPET study.§

§ These data have not been presented to the FDA for evaluation. Information from the NuProtect study is presented in parallel to the SIPPET study for context, but please note that these trials were performed under different conditions and with different populations.
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NUWIQ offers convenient and flexible dosing for children and adults.

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References
  1. NUWIQ full Prescribing Information. Paramus, NJ: Octapharma USA, Inc.; rev 2017.
  2. Data on file. Paramus, NJ: Octapharma USA, Inc; 2015.
  3. Valentino LA, et al. Haemophilia. 2015;21:1-12.
  4. Kessler C, et al. Haemophilia. 2015;21(Suppl. 1): 1-12.
  5. Peyvandi F, et al. N Engl J Med. 2016;374:2054-2064.
  6. Ri Liesner, Ellis J. Neufeld; Inhibitor Development with Simoctocog Alfa in Previously Untreated Patients with Severe Haemophilia a: Final Results of the Nuprotect Study. Blood 2019; 134 (Supplement_1): 903. doi: https://doi.org/10.1182/blood-2019-125520