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Australian prescription medicine decision summary
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.
Summary of submission
|Active ingredient||Nonacog beta pegol|
|Anatomical Therapeutic Chemical (ATC) code||B02BD04|
|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|
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)|
|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.
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 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?
The following table summarises the key steps and dates for this application.
|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.