In a prospective, open-label, international, multi-centre Phase III study (GENA-08) 32 adult PTPs with severe hemophilia A received Nuwiq® prophylaxis. Of the 32 subjects, 24 had at least 50 EDs and were followed for at least 6 months. Thirty-one subjects accumulated more than 50 EDs and the same 31 subjects stayed in the study for at least 176 days. The mean prophylactic dose was 32.8 IU/kg and was administered every other day. In the pivotal study, 50% of patients experienced no bleeding. The overall efficacy of prophylactic treatment was evaluated based on the rate of bleeding episodes (BEs).
ADULT STUDIES
Nuwiq®: Prophylactic Treatment Assessed in a Phase III, Prospective, Non-controlled, Open-label Study (GENA-08)
| Spontaneous BEs | 0 (0 – 8.6) |
| Traumatic BEs | 0 (0 – 8.3) |
| All BEs | 0.90 (0 – 14.7) |
Data are median, range.
BE = Bleeding episode
Adapted from Nuwiq Product Monograph
Nuwiq®: Efficacy in the Treatment of Breakthrough BEs During Prophylaxis Assessed in a Phase III, Prospective, Non-controlled, Open-label study (GENA-08)
- The efficacy of Nuwiq® in the treatment of breakthrough BEs during prophylaxis was assessed in a total of 32 previously treated adults with severe hemophilia A (GENA-08).
- The overall efficacy of on-demand treatment was evaluated based on criteria including improvement of objective signs of bleeding, number of infusions required to control the bleeding, and the time until bleeding improvement.
- These patients experienced a total of 30 BEs which were treated with Nuwiq®. Efficacy was rated excellent or good in 100% of these patients
- Of the 30 BEs, 88.9% were treated with one or two infusions. The mean dose of Nuwiq® per infusion for the treatment of breakthrough BEs was 33.3 IU/kg.
Efficacy of Nuwiq® in the treatment of bleeding episodes, number of infusions used to treat BEs and doses per infusion in clinical study GENA-08
| Efficacy rating | GENA-08 (n = 32 subjects) |
|---|---|
| Number of BEs | 44 |
| Number of treated BEs | 30 |
| Any BE (N) | 28* |
| Excellent | 71.4% |
| Good | 28.6% |
| Moderate | – |
| None | – |
| Number of infusions Median (range) | 1.0 (1-12) |
| Dose per infusion (IU/kg) Mean ± SD (range) | 33.3 ± 6.7 (20-53) |
Efficacy rating data are percentages. * For 2 BEs, no efficacy assessments were available.
BE = bleeding episode; N = number of BEs.
Adapted from Nuwiq Product Monograph
Design: Prospective, non-controlled, open-label, multinational, multi-centre, Phase III study
Dosing: IVR: 50 IU/kg
Prophylactic treatment: 30–40 IU FVIII/kg every other day or 3 times weekly. Two dose escalations of +5 IU/kg each allowed in case of an inadequate response (>2 spontaneous BEs during 1 month)
Treatment of BEs:
Minor BEs:
20–30 IU FVIII/kg every 12–24 h until BE resolution
Moderate to major BEs:
30–40 IU FVIII/kg every 12–24 h until BE resolution
Major to life threatening BEs:
Initial dose of 50–60 IU FVIII/kg and then a dose of 20–25 IU FVIII/kg every 8–12 h until BE resolution
Duration: >50 EDs and >6 months
Participants: 32 male PTPs with severe hemophilia A, age 37.3 years (mean), (range of 18-75)
safety information
Nuwiq® is indicated for the treatment and prophylaxis of bleeding in patients of all ages suffering with hemophilia A (congenital factor VIII deficiency).
Nuwiq® is also indicated for the peri-operative treatment of hemophilia A patients.
Pediatrics (< 12 years of age): Half-life (T1/2) and incremental in vivo recovery (IVR) are slightly lower in children than in adults and clearance is slightly higher. Efficacy in prophylaxis and the treatment of bleeds is comparable between children and adults. Data indicate that Nuwiq® is equally efficacious and safe in this population.
Nuwiq® is contraindicated for patients who are hypersensitive to the active substance or to any ingredient in the formulation or component of the container.
- It is essential to confirm the deficiency of FVIII before starting treatment with Nuwiq®.
- Nuwiq® powder should be reconstituted according to directions and using only the supplied solvent (2.5 mL water for injection) and the supplied injection set to ensure optimal effectiveness and safety.
- Transmission of infectious viruses can result from use of contaminated needles or accidental puncture with contaminated needles.
- Strongly recommended that every time Nuwiq® is administered to a patient, the name and batch number of the product are recorded in order to maintain a link between the patient and the batch of the product.
- All patients treated with Nuwiq® should be carefully monitored for the development of inhibitors by appropriate clinical observations and laboratory tests
Allergic-type hypersensitivity reactions are possible. - Risk of central venous access device (CVAD)-related complications.
- Do not use this medicine after the expiry date stated on the label or if signs of deterioration of the tamper-proof packaging, especially of the syringe and/or the vial, are visible.
- Pregnant and Nursing Women: animal reproduction studies have not been conducted with Nuwiq® and it has not been used in women. Nuwiq® should be used during pregnancy and lactation only if clearly indicated.
Please consult the product monograph at https://www.octapharma.ca/en/therapies/product-overview/ for important information relating to adverse reactions, drug interactions, and dosing information which have not been discussed on this site. The product monograph is also available by calling us at 1-888-438-0488 or by contacting us via email: medinfo@octapharma.ca
Health professionals may report adverse reactions to adr.canada@octapharma.com. You can also report any suspected Adverse Reactions directly to the Canada Vigilance Program (https://www.canada.ca/en/health-canada/services/drugs-health-products/medeffect-canada/adverse-reaction-reporting.html).
