Product

Nuwiq® (human coagulation factor VIII recombinant DNA, simoctocog alfa)

 

Human coagulation factor VIII (rDNA), simoctocog alfa is a purified protein. Nuwiq® is produced by recombinant DNA technology in genetically modified human embryonic kidney (HEK) cells. No animal or human derived materials are added during the manufacturing process or to the final medicinal product.

Powder and solvent for solution for injection available in vials of 

  • 250 IU
  • 500 IU
  • 1000 IU
  • 2000 IU
 

Reconstitution

Reconstitution guide

This video shows patients how to prepare an injection of Nuwiq® effectively and safely. It is to be used alongside guidance given by the local Haemophilia Centre when the patient was trained to use this medicine. This video should also be used alongside information provided in Nuwiq® packs. 

 

 

Making Nuwiq® Film

How we make Nuwiq®

Glossary for the film

Download

 

Product

FAQs

What is Nuwiq®?

Nuwiq® is an injectable medicine that contains the active substance human recombinant coagulation factor VIII (simoctocog alfa). 

What are the therapeutic indications of Nuwiq®?

Nuwiq® is licensed for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency). Nuwiq® can be used for all age groups.

What vial sizes does Nuwiq® come in, and what is the reconstituted volume?

Nuwiq® is supplied in packs containing single-use vials of 250, 500, 1000 or 2000 International Units (IU). Packs can be distinguished by colour-coded printing on the label and the box. The reconstituted volume is 2.5 ml for all vial sizes.

How is Nuwiq® produced?

Nuwiq® is produced by recombinant DNA technology in genetically modified human embryonic kidney (HEK) cells. No animal or human derived materials are added during the manufacturing process or to the final medicinal product. The process also includes a solvent-detergent viral inactivation step and a 20 nm filtration step. A short film of the manufacturing process for Nuwiq® is available here.

What is the content of a single Nuwiq® pack?

Nuwiq® is available in vials containing 250, 500, 1000 or 2000 International Units (IU) of human coagulaton factor VIII (rDNA), simoctocog alfa. Each pack of Nuwiq® contains:

• Powder in a glass vial, closed with a stopper and sealed with a flip-off cap
• Water for Injections in a pre-filled syringe
• A sterile vial adapter for reconstitution with one butterfly needle and two alcohol swabs

• A package leaflet with information for the user

What is the shelf life of Nuwiq® and how should it be stored?

The shelf-life is 2 years when stored in a refrigerator (2°C – 8°C).

During the shelf-life, the product may be kept at room temperature (up to 25°C) for a single period not exceeding 1 month. Once the product has been taken out of the refrigerator the product must not be returned to the refrigerator. Please record the beginning of storage at room temperature on the product carton.

Do not freeze.

Keep the vial in the outer carton in order to protect from light.
After reconstitution, chemical and physical in-use stability has been demonstrated for 24 hours when stored at room temperature. Do not refrigerate after reconstitution. From a microbiological point of view, the product should be used immediately after reconstitution. If not used immediately, in-use storage times and conditions prior to use are the responsibility of the user.

 

 

 

 

Clinical

FAQs

Which clinical studies with Nuwiq® have been completed and reported results?

 

Nuwiq® has been assessed in three pivotal multicentre, multinational studies (GENA-011, GENA-033 and GENA-084).

 

- The completed (GENA-01) and (GENA-08) studies evaluated the pharmacokinetics, safety, immunogenicity and efficacy of Nuwiq® in the treatment and prophylaxis of bleeding in 54 previously treated adult and adolescent patients with severe haemophilia A.

- A completed study (GENA-03) investigated the pharmacokinetics, efficacy, safety and immunogenicity of Nuwiq® in 59 previously treated children aged 2-12 years with severe haemophilia A.

What is the efficacy of Nuwiq® in on-demand treatment?

Treatment of 986 bleeding episodes was rated "excellent or good" in 94.4% of cases1, and 96.7% of all bleeds were managed with one or two infusions2. The effectiveness of Nuwiq® at controlling bleeding episodes was assessed after each infusion and at the end of a bleeding episode by the patient, or patient and physician jointly.

What is the efficacy of Nuwiq® during prophylactic treatment of adult patients?

A clinical trial (GENA-084) involved 32 adult patients receiving Nuwiq® as bleed prophylaxis every other day. Efficacy of prophlyaxis was assessed after a total of at least 50 exposure days per subject at the end of the study at six months. The frequency of spontaneous breakthrough bleeds/month during prophylactic treatment was 0.188 ± 0.3074. There was no major or life threatening bleeding, and 50% of patients (16/32) did not experience any bleeding episodes during prophlyaxis with Nuwiq®2.

What is the efficacy of Nuwiq® during prophylactic treatment of children?

During a paediatric study, GENA-033, the annualised bleeding rate in children undergoing prophylaxis was 4.1, comprising 2.6 bleeds in the younger children (two to five years) and 5.6 bleeds in the older children (six to 12 years). There were no major or life-threatening bleeds2.

How has Octapharma investigated inhibitors with Nuwiq®

The occurrence of inhibitors have been investigated during the registration studies. A total of 135  PTPs with severe haemophilia A, comprising 74 adults and 61 children1,3-5 received Nuwiq®. None of these patients developed an inhibitor2. Octapharma is running a study which began in 2013 and aims to recruit 100 PUPs6. The trial will assess the immunogenicity, efficacy, safety and tolerability of Nuwiq®.

How has Octapharma investigated personalised prophylaxis with Nuwiq®?

GENA-2110 (NuPreviq) is a prospective, open-label, multicentre study to assess the efficacy and safety of individually tailored prophylaxis with Nuwiq® in adult previously treated patients (PTPs) with severe haemophilia A. The goal of this study is to provide individualised treatment for each patient based on their own pharmacokinetic profiles.

Will Octapharma be providing any service to support personalised prophylaxis?

Octapharma will look to create a service for patients based on the methodology of the GENA-21 (NuPreviq) study.

 

Clinical

The first recombinant FVIII product of human-cell origin licensed in Europe

 

References

References

1. ClinicalTrials.gov (2014) Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A [Online] Available at http://clinicaltrials.gov/show/NCT00989196 (Accessed 13 October 2014).

2. Valentino L.A. et al. (2014) 'The first recombinant FVIII produced in human cells - an update on its clinical development programme', Haemophilia, vol. 20 (Suppl. 1), pp. 1-9.

3. EU Clinical Trials Register (2014) Prospective clinical study in children with severe haemophilia A to investigate clinical efficacy, immunogenicity, pharmacokinetics, and safety of human-cl rh FVIII [Online] Available at https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-018644-14/GB (Accessed 13 October 2014).

4. ClinicalTrials.gov (2014) Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A [Online] Available at http://clinicaltrials.gov/show/NCT01125813 (Accessed 13 October 2014).

5. International Standard Randomised Controlled Trial Number Register (2014) Clinical study to investigate the pharmacokinetics, efficacy, safety and immunogenicity of human-cl rhFVIII in previously treated patients with severe haemophilia A [Online] Available at http://www.controlled-trials.com/ISRCTN87293301 (Accessed 13 October 2014).

6. ClinicalTrials.gov (2014) Human-cl-rhFVIII in Previously Untreated Patients [Online] Available at http://clinicaltrials.gov/show/NCT01712438 (Accessed 13 October 2014).

7. Summary of Product Characteristics for Nuwiq® available at https://www.medicines.org.uk/emc/medicine/29373.

8. Casademunt E. et al. (2012) ‘The first recombinant human coagulation factor VIII of human origin: human cell line and manufacturing characteristics’, European Journal of Haematology, vol. 89(2), pp. 165-76.

9. Sandberg H. et al. (2013) ‘Functional characteristics of the novel, human-derived recombinant FVIII protein product, human-cl rhFVIII’, Thrombosis Research, vol. 130, pp. 808-17.

10. EU Clinical Trials Register (2014) Prospective, open-label, multicentre phase 3b study to assess the efficacy and safety of individually tailored prophylaxis with Human-cl rhFVIII in previously treated adult patients with severe haemophilia A [Online] Available at https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-001556-35/GB (Accessed 13 October 2014).

11. http://www.ema.europa.eu/ema/

12. Kannicht C. Ramström M, Kohla G et al. (2013). Characterisation of the post-translational modifications of a novel, human cell line-derived recombinant human factor VIII. Thromb Res; 131:78-88.

13. Shen BW, Spiegel PC, Chang CH, et al. (2008). The tertiary structure and domain organization of coagulation factor VIII. Blood; 111: 1240-1247.

14. Sadler JE (1998). Biochemistry and genetics of von Willebrand factor. Annu Rev Biochem; 67:395-424.

15. Tiede A. et al. (2014). Personalized prophylaxis with human-cl rhFVIII in adult patients with severe haemophilia A. Poster presented at the Bari International Conference, Bari, Italy, 3-5 October 2014.

 

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Details of clinical trials sponsored by Octapharma are provided at clinicaltrials.gov and clinicaltrialsregister.eu


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