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Australian prescription medicine decision summary

10 September 2019

The Australian Prescription Medicine Decision Summary provides a short overview of the TGA's evaluation process leading to the registration of a new prescription medicine on the Australian Register of Therapeutic Goods (ARTG).

More in-depth information about the evaluation will be available in the Australian Public Assessment Report (AusPAR) for a particular prescription medicine, which can be found on the AusPAR search page once published.

Product name Refixia
Active ingredient Nonacog beta pegol
Anatomical Therapeutic Chemical (ATC) code B02BD04
Decision Approved
Date of decision 2 September 2019
Date of entry onto ARTG 4 September 2019
ARTG number(s) 308424, 308425, 308426
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
Sponsor's name and address

Novo Nordisk Pharmaceuticals Pty Ltd

PO Box 7586, Baulkham Hills NSW 2153

Dose form(s) Powder and solvent for injection
Strength(s) 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).

Container(s) Vial (powder) and prefilled syringe (solvent)
Pack size(s) 1
Route(s) of administration Intravenous

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.

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).

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.

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.

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

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.

Further information

The latest Product Information (PI) and Consumer Medicines Information (CMI) can be found at: ARTG search.

Australian Public Assessment Reports (AusPARs) can be found at: AusPAR search.

The latest news and updates regarding therapeutic goods regulation can be found at: TGA news room.