Consumer comments close Wednesday, 16 September 2026.
The Pharmaceutical Benefits Advisory Committee (PBAC) will consider an application to list sepiapterin (Sephience®) on the Pharmaceutical Benefits Scheme (PBS) at its November 2026 meeting.
Sepiapterin, used in combination with a low-phenylalanine diet, is being considered for the treatment of hyperphenylalaninaemia (HPA) due to phenylketonuria (PKU).
The proposed eligibility criteria include:
- patients less than one month of age with a baseline blood phenylalanine level above 360 micromol/L; or
- patients more than one month of age with a baseline blood phenylalanine level above 600 micromol/L.
The proposed listing is Authority Required (Streamlined).
Why your voice matters
Consumer comments help the PBAC understand the lived experience of PKU, the impact of current dietary management and treatment options, and what access to an additional treatment could mean for individuals and families.
We encourage people living with PKU, as well as family members and carers, to contribute—even if you or your family member may not meet the proposed eligibility criteria. Your experience can provide valuable insight into the broader impact of PKU and the importance of having access to a range of treatments and management tools.
You do not need to be an expert or use technical language. Sharing your own experience—what managing PKU involves, the challenges you face and what another treatment option could mean for you or your family—is what matters.
MDDA’s submission
MDDA will also prepare a community submission addressing:
- the restrictive and demanding nature of managing a low-phenylalanine diet
- the impact of current management and treatment options on individuals and families
- findings from MDDA’s recent quality-of-life and PKU community surveys
- the broader health and wellbeing impacts associated with PKU
- the potential benefits of access to treatment and the importance of individual treatment choice.
How to have your say
Visit the [PBAC public consultation page] and select Sepiapterin (Sephience®) from the drop-down menu at Question 7.
Making a comment is straightforward, and the survey questions will guide you through the information you may wish to provide.
Consumer comments close Wednesday, 16 September 2026.
MDDA information webinar
MDDA will hold an information webinar with a clinician to help our community understand sepiapterin, the proposed PBS listing and the consumer comment process.
The webinar date and registration details will be shared shortly.