PEGVALIASE
Information current as at: 1 September 2026
Submission Details
- Brand name:
-
- Palynziq®
- Form and strength:
-
Injection 2.5 mg in 0.5 mL pre-filled syringe
Injection 10 mg in 0.5 mL pre-filled syringe
Injection 20 mg in 1 mL pre-filled syringe - Submission sponsor:
- BIOMARIN PHARMACEUTICAL AUSTRALIA PTY LTD
- Condition/indication:
(therapeutic use) -
- Phenylketonuria (PKU)
- Listing requested:
- Resubmission to request listing of pegvaliase for the treatment of patients aged 16 years and older with PKU who have inadequate blood phenylalanine control (baseline blood phenylalanine level above 600 micromoles per L) despite prior management with available treatment options (including a phenylalanine restricted diet and sapropterin). An inadequate response to a trial of sapropterin is defined as failure to achieve a 30 per cent or greater reduction in blood phenylalanine from baseline following initial treatment with sapropterin. The submission also seeks PBAC consideration for extending eligibility to patients aged 16 years and older with PKU who have a protein tolerance of less than 15 grams per day, even if sapropterin responsive. This item was previously considered by the PBAC at its March 2026 meeting.
- Funding program:
- PBS General Schedule
- Request authority level:
- Authority Required (Telephone/Online)
- PBAC Submission type:
- New PBS listing (Matters arising/Matters outstanding)
- Comment:
- --
- Other PBAC consideration:
Progress Details
-
Submission received for: - July 2026 PBAC meeting
-
Opportunity for consumer comment: - Open 19/11/2025 and close 21/01/2026 (see PBS Website)
-
PBAC meeting: - Held on 08/07/2026
-
5Lodgement of required documentation:
- 17/08/2026
-
Acceptance of complete documentation:
- Under consideration
-
6Agreement to listing arrangements:
- Has not yet commenced
-
7Government processes:
- Has not yet commenced
-
8Medicine listed on the PBS:
- Has not yet occurred
PBAC Outcome
The PBAC recommended pegvaliase for the treatment of patients with phenylketonuria (PKU) who have inadequate blood phenylalanine (Phe) control despite prior treatment with a Phe-restricted diet and sapropterin.
In March 2026, the PBAC had deferred making a recommendation for pegvaliase to allow further consultation with the sponsor and Department regarding how best to implement the intent of the sponsor’s pricing structure and financial arrangement proposal. At that time, the PBAC considered it was unclear how the proposed pricing structure and financial arrangement would achieve a cost-effective price for pegvaliase and adequately manage the financial risk associated with listing pegvaliase on the PBS.
The PBAC considered that the listing proposal, submitted in response to the March 2026 deferral, provided increased confidence that the intended level of cost-effectiveness could be achieved and the financial risk to the Commonwealth would be appropriately managed.
The PBAC acknowledged the challenges of living with PKU including the severe and lifelong burden of dietary restrictions and the cognitive impacts of high Phe levels including difficulty concentrating, reduced mental clarity, anxiety and fatigue. The PBAC reaffirmed its view that there is a high unmet clinical need for effective treatments in the proposed patient population.
The PBAC maintained its view from its March 2026 consideration of pegvaliase, that pegvaliase is more effective than a Phe-restricted diet alone at reducing blood Phe levels. The PBAC also recalled that pegvaliase was associated with higher rates of hypersensitivity and skin reactions compared with a Phe- restricted diet alone.
The PBAC also considered that it would be reasonable to flow-on changes to all PBS listings of sapropterin, to allow nurse practitioner prescribing in consultation with a metabolic physician in the initial treatment and prevent concomitant use of pegvaliase and sapropterin for just this indication.