ADULTS & ADOLESCENTS studies

Nuwiq^®: In the GENA-01 study, efficacy of on-demand treatment (secondary endpoint) was assessed in adults and adolescents ages 12-65 years

Nuwiq^®:Efficacy of on-demand treatment assessed in adults and adolescents

A total of 986 BEs was treated with Nuwiq in a prospective, randomized, actively controlled, cross-over, open-label, Phase II study of 22 adult and adolescent PTPs with severe hemophilia A. In total, 416 (42.2%) were minor, 566 (57.4%) were moderate to major and 3 (0.3%) were major to life-threatening. The severity for one BE was unknown. The mean on-demand treatment dose was 32.3 IU/kg. The median (range) number of infusions required to stop a BE was 1.0 (range 1–13). The mean duration of treatment of BEs overall was 1.1 days. 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. The proportion of BEs with successful treatment (rated as “good” or “excellent”) was 94.4% (931/986 BEs). The rate of BEs successfully treated with just 1 or 2 infusions was 96.8% (954/986 BEs).

Efficacy of Nuwiq® in the treatment of bleeding episodes, number of infusions used to treat BEs and doses per infusion in clinical study GENA-01

Efficacy rating	GENA-01 (n = 22 subjects)
Number of BEs	997
Number of treated BEs	986
Any BE (N)	986
Excellent	60.3%
Good	34.1%
Moderate	5.5%
None	–
Number of infusions Median (range)	1.0 (1-13)
Dose per infusion (IU/kg) Mean ± SD (range)	32.3 ± 10.6 (7-61)

Efficacy rating data are percentages.
BE = bleeding episode; N = number of BEs.
Adapted from Nuwiq Product Monograph

Study Parameters (GENA-01)

Design: Prospective, randomized, actively controlled, cross-over, open-label, multi-centre, Phase II study

Dosing: PK and IVR: 50 IU/kg

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: >6 months and >50 EDs (the study was clinically completed by end of September 2012 regardless of the number of EDs)

Participants: 22 male PTPs with severe hemophilia A, age 39.6 years (mean), (range of 12–65) includes 2 adolescents aged 12 and 14 years

safety information

Indications and clinical use

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.

Contraindications

Nuwiq® is contraindicated for patients who are hypersensitive to the active substance or to any ingredient in the formulation or component of the container.

Relevant warnings and precautions

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.

For more information

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

Adverse Event Reporting

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).