Esperoct
Registration timeline
The following table summarises the key steps and dates for this comparable overseas regulator approach B (COR-B) application.
|
Description |
Date |
|
Submission dossier accepted and first round evaluation commenced
|
3 June 2022 |
|
First round evaluation completed |
30 August 2022 |
|
Sponsor provides responses on questions raised in first round evaluation |
21 October 2022 |
|
Second round evaluation completed |
6 March 2023 |
|
Delegate’s Overall benefit-risk assessment |
29 March 2023 |
|
Sponsor’s pre-Advisory Committee response |
Not applicable |
|
Advisory Committee meeting |
Not applicable |
|
Registration decision (Outcome) |
31 May 2023 |
|
Completion of administrative activities and registration on ARTG |
5 June 2023 |
|
Number of working days from submission dossier acceptance to registration decision* |
144 |
* The COR-B process has a 175 working day evaluation and decision timeframe.
Calcium chloride dihydrate, histidine, hydrochloric acid, methionine, polysorbate 80, sodium chloride, sodium hydroxide, sucrose, and water for injections.
Treatment should be initiated under the supervision of a physician experienced in the treatment of haemophilia.
The dose, dosing interval and duration of substitution therapy depend on the severity of the factor VIII deficiency, the location and extent of bleeding, the targeted factor VIII activity level and the patient’s clinical condition.
For further information refer to the Product Information.
Drugs 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.
Esperoct (turoctocog alfa pegol) was approved for the following therapeutic use:
Esperoct, is a long-acting recombinant Factor VIII concentrate indicated for use in previously treated patients with haemophilia A for:
- Routine prophylactic treatment to prevent or reduce the frequency of bleeding episodes
- On-demand treatment and control of bleeding episodes
- Peri-operative management of bleeding (surgical prophylaxis)
Esperoct does not contain von Willebrand factor, and therefore is not indicated in patients with von Willebrand’s disease.
Haemophilia A is a X-chromosomal hereditary disorder of blood coagulation due to decreased levels or absence of factor VIII:C that results in bleeding into joints, muscles or internal organs, either spontaneously or as a result of accidental or surgical trauma.
Turoctocog alfa pegol is a purified recombinant human factor VIII (rFVIII) product with 40 kDa polyethylene glycol (PEG), which is conjugated to protein. The PEG is attached to the O-linked glycan in the truncated B domain of rFVIII (turoctocog alfa). The mechanism of action of turoctocog alfa pegol is based on the substitution of inadequate or absent factor VIII in patients with haemophilia A.
When turoctocog alfa pegol is activated by thrombin at the injury site, the a3 region and the B domains containing the PEG are cleaved off, producing active recombinant factor VIII (rFVIIIa), which is similar in structure to native factor VIIIa.
This replacement therapy raises factor VIII plasma levels, temporarily correcting the factor VIII deficiency and bleeding tendency.
- Esperoct (turoctocog alfa pegol) is to be included in the Black Triangle Scheme. The PI [Product Information] and CMI [Consumer Medicines Information] for Esperoct 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 Esperoct EU [European Union] -risk management plan (RMP) (version 2.1, dated 14 August 2022, data lock point 13 October 2021), with Australia specific annex (version 0.2, dated 12 October 2022), included with Submission PM-2022-01578-1-6, 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 the 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 (revision 1), Part VII.B Structures and processes. Note that submission of a PSUR does not constitute an application to vary the registration.
- Laboratory testing & compliance with Certified Product Details (CPD):
- All batches of Esperoct supplied in Australia must comply with the product details and specifications approved during evaluation and detailed in the Certified Product Details (CPD).
- When requested by the TGA, the Sponsor should be prepared to provide product samples, specified reference materials and documentary evidence to enable the TGA to conduct laboratory testing on the Product. Outcomes of laboratory testing are published biannually in the TGA Database of Laboratory Testing Results http://cwww.tga.gov.au/ws-labs-index and periodically in testing reports on the TGA website.
- The Certified Product Details (CPD), as described in Guidance 7: Certified Product Details of the Australian Regulatory Guidelines for Prescription Medicines (ARGPM), in PDF format, for the above products should be provided upon registration of these therapeutic goods. In addition, an updated CPD should be provided when changes to finished product specifications and test methods are approved in a Category 3 application or notified through a self-assessable change.
A template for preparation of CPD for biological prescription medicines can be obtained from the TGA website
[for the form] https://www.tga.gov.au/form/certified-product-details-cpd-biologicalprescription- medicines
[for the CPD guidance] https://www.tga.gov.au/resources/resource/guidance/guidance-7-certified-product-details
- The final report for clinical trial NN7088-3908 in previously untreated patients should be submitted to the TGA within 6 months of completion.
- For all injectable products the Product Information must be included with the product as a package insert.