Pegvaliase (Palynziq) Consumer Comments – Close 21 January 2026
The Pharmaceutical Benefits Advisory Committee will consider pegvaliase (Palynziq) for the second time in March 2026 for the following proposed populations:
| 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. |
The addition of a population who have a natural protein tolerance of less than 15g per day was added after feedback from patients, MDDA and clinicians about unmet need for people who may be sapropterin responsive but still may experience a very restricted diet. Feedback from the community about an appropriate restriction was gathered through a community forum and survey.
Consumer Comments are open now until January 21 and this is your chance to have input into the decision. We encourage you to provide comment even if you or your family member may not be eligible for this treatment. Support from the community will help to gain access for those with a high level of need.
MDDA will comment on:
- The restrictive nature of managing a restriction of 15 grams or less per day
- The burden on individuals and family members of current treatment options
- The input we received from the community about the proposed restriction
- The information we gained from the last two surveys we have conducted on quality of life and PKU
- PKU and common co-morbidities
- The benefits of access to treatment and the importance of people having access to a range of treatments and tools to help manage PKU
Use this link to provide your input, when you do the survey select Pegvaliase (Palynziq) from the drop down menu.
Making a comment is straightforward and the survey questions guide you as to what to put in your comment.
MDDA has also prepared some information to help:
Click here to see a presentation by Prof. Cary Harding M.D., Professor of Molecular and Medical Genetics, School of Medicine
about pegvaliase.
Click here to see a presentation by Louise Healy, MDDA Vice President and Education and Advocacy Manager, Rare Voices Australia on making a consumer comment.
This tip sheet from the Patient Voice Initiative may also help prompt you about what to talk about.
Comments close 21 January 2026