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Refixia

Sponsor
Novo Nordisk Pharmaceuticals Pty Ltd
ARTGs
Device/Product name
Refixia
Active Ingredient
Nonacog beta pegol
Date of decision
Published
Submission type
New biological entity
ATC codes
B02BD04
Decision
Approved
What was the decision based on
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Refixia was considered favourable for the therapeutic use approved.
What steps were involved in the decision process

Registration timeline

The following table summarises the key steps and dates for this application.

Description Date
Submission dossier accepted and first round evaluation commenced 28 September 2018
First round evaluation completed 28 February 2019
Sponsor provides responses on questions raised in first round evaluation 3 May 2019
Second round evaluation completed 20 June 2019
Delegate's overall benefit-risk assessment and request for Advisory Committee advice 1 July 2019
Sponsor's pre-Advisory Committee response 12 July 2019
Advisory Committee meeting 1-2 August 2019
Registration decision (Outcome) 2 September 2019
Completion of administrative activities and registration on ARTG 4 September 2019
Number of working days from submission dossier acceptance to registration decision* 192

*Statutory timeframe for standard applications is 255 working days

Date of entry onto ARTG
Original publication date
Black triangle scheme
Yes. This product will remain in the scheme for 5 years, starting on the date the product is first supplied in Australia.
Dose forms
Powder and solvent for injection
Strength
500 IU, 1000 IU, 2000 IU
Other ingredients

Powder: Sodium chloride, Histidine, Sucrose, Polysorbate 80, Mannitol, Sodium hydroxide (for pH adjustment), Hydrochloric acid (for pH adjustment).

Solvent: Histidine, Water for injection, Sodium hydroxide (for pH adjustment), Hydrochloric acid (for pH adjustment).

Containers
Vial (powder) and prefilled syringe (solvent)
Pack sizes
1
Routes of administration
Intravenous
Dosage

Routine prophylaxis: 40 IU/kg body weight once weekly.

Bleeding episodes: The dose and duration of the replacement therapy depends on the location and severity of the bleeding.

For further information refer to the Product Information.

Pregnancy category
Category B2Drugs which have been taken by only a limited number of pregnant women and women of childbearing age, without an increase in the frequency of malformation or other direct or indirect harmful effects on the human fetus having been observed.Studies in animals are inadequate or may be lacking, but available data show no evidence of an increased occurrence of fetal damage.The use of any medicine during pregnancy requires careful consideration of both risks and benefits by the treating health professional. This must not be used as the sole basis of decision making in the use of medicines during pregnancy. The TGA does not provide advice on the use of medicines in pregnancy for specific cases. More information is available from obstetric drug information services in your State or Territory.
What was approved

Refixia (nonacog beta pegol) was approved for the following therapeutic use:

Treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia B (congenital factor IX deficiency).
What is this medicine and how does it work
Refixia is a purified recombinant human factor IX (rFIX) with a 40 kDa polyethylene-glycol (PEG) conjugated to the protein.Patients with haemophilia B are deficient in coagulation factor IX, which is required for effective haemostasis. The administration of Refixia increases plasma levels of factor IX and can temporarily correct the coagulation defect in haemophilia B patients.Factor IX is activated by factor XIa and by factor VII/tissue factor complex. Upon activation of Refixia, the activation peptide including the 40 kDa polyethylene-glycol moiety is cleaved off, leaving the native factor IX molecule. Activated factor IX, in combination with activated factor VIII, activates factor X. Activated factor X converts prothrombin into thrombin. Thrombin then converts fibrinogen into fibrin and a clot is formed.
What post-market commitments will the sponsor undertake

The following specific conditions of registration apply to this approval:

  • Refixia (nonacog beta pegol) is to be included in the Black Triangle Scheme. The Product Information (PI) and Consumer Medicines Information (CMI) for Refixia must include the black triangle symbol and mandatory accompanying text for five years, which starts from the date that the sponsor notifies the TGA of supply of the product.
  • The Refixia (nonacog beta pegol) European Union-Risk Management Plan (EU-RMP) (version 4.0, dated 28 April 2017, data lock point 1 January 2016), with Australian Specific Annex (version 0.3, dated 2 April 2019), included with submission PM-2018-02720-1-6, and any subsequent revisions, as agreed with the TGA will be implemented in Australia.

    An obligatory component of risk management plans is routine pharmacovigilance. Routine pharmacovigilance includes the submission of periodic safety update reports (PSURs).

    Reports are to be provided in line with the current published list of EU reference dates and frequency of submission of PSURs until the period covered by such reports is not less than three years from the date of this approval letter.

    The reports are to at least meet the requirements for PSURs as described in the European Medicines Agency's Guideline on good pharmacovigilance practices (GVP) Module VII-periodic safety update report (Rev 1), Part VII.B Structures and processes. Note that submission of a PSUR does not constitute an application to vary the registration.

  • For all injectable products the Product Information must be included with the product.

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