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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.
Australian prescription medicine decision summary
|Submission type|| |
New biological entity
|Product name|| |
|Active ingredients|| |
|ATC codes|| |
|Date of decision|| |
6 July 2021
|Date of entry onto ARTG|| |
14 July 2021
|Original publication date|| |
22 July 2021
|ARTG numbers|| |
341752, 341753 and 341754
|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
BioMarin Pharmaceutical Australia Pty Ltd
|Sponsor address|| |
119 Willoughby Road, Crows Nest NSW 2065
|Dose forms|| |
Solution for injection
2.5 mg/0.5 mL, 10 mg/0.5 mL and 20 mg/mL
|Other ingredients|| |
Trometamol, trometamol hydrochloride, sodium chloride, cinnamic acid and water for injections
|Pack sizes|| |
1 (applicable to all strengths) and 10 (only applicable to 20 mg/mL syringes)
|Routes of administration|| |
Treatment with Palynziq should be directed by physicians experienced in the management of phenylketonuria (PKU) and in the context of a multidisciplinary team, including dietician.
Dosage is based on blood phenylalanine level of the patient.
For further information refer to the Product Information.
|Pregnancy category|| |
Drugs which have caused, are suspected to have caused or may be expected to cause, an increased incidence of human fetal malformations or irreversible damage. These drugs may also have adverse pharmacological effects. Accompanying texts should be consulted for further details.
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.
Palynziq (pegvaliase) was approved for the following therapeutic use:
Palynziq is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control despite prior management with available treatment options.
The following table summarises the key steps and dates for this comparable overseas regulator approach B (COR-B) application.
|Designation, orphan||15 April 2020|
|Submission dossier accepted and first round evaluation commenced||15 April 2020|
|First round evaluation completed||12 February 2021|
|Sponsor provides responses on questions raised in first round evaluation||12 March 2021|
|Second round evaluation completed||12 April 2021|
|Delegate's overall benefit-risk assessment and request for Advisory Committee advice||3 May 2021|
|Sponsor's pre-Advisory Committee response||17 May 2021|
|Advisory Committee meeting||3 and 4 June 2021|
|Registration decision (Outcome)||6 July 2021|
|Completion of administrative activities and registration on ARTG||14 July 2021|
|Number of working days from submission dossier acceptance to registration decision*||166|
*The COR-B process has a 175 working day evaluation and decision timeframe.
- Palynziq (pegvaliase) is to be included in the Black Triangle Scheme. The Product Information and Consumer Medicines Information for Palynziq 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 Palynziq European Union (EU)-risk management plan (RMP), version 3.0, dated 6 October 2020, data lock point 17 April 2019), with Australian Specific annex (version 0.2, dated 5 March 2021) included with Submission PM-2020-04119-1-3, and any subsequent revisions, as agreed with the TGA will be implemented in Australia.
An obligatory component of RMP 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 (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 as a package insert.