MDDA Announcement:

Ensuring treatment access for all Australians with PKU

30 March 2022

Today (Wednesday, 30 March), the Pharmaceutical Benefits Advisory Committee (PBAC) published the agenda for its next meeting, which will be held from 6-8 July. Two treatments for PKU will be included on the July PBAC Meeting Agenda, and will be considered by the PBAC for listing on the Pharmaceutical Benefits Scheme (PBS).

The two treatments that will be considered are the oral medicine, Kuvan (sapropterin dihydrochloride), and the new subcutaneous (injected under the skin) medicine, Palynziq (pegvaliase). Both medicines will be reviewed as potential treatment options for Australian adults living with PKU. Kuvan is currently listed on the PBS for people with PKU aged under 18 years (since 1 May 2019), and for pregnant women/maternal PKU (since 1 July 2021). As a new treatment, Palynziq will be reviewed for listing on the PBS for the first time, for those aged 16 years and over.

MDDA is pleased that our ongoing advocacy regarding the high levels of unmet medical need faced by adults with PKU, and the inequity in treatment access for this group, will be reviewed by the PBAC in July. As many of you would be aware, the only currently available treatment option for adults with PKU is to adhere to a strict, lifelong, low-protein diet. This is despite research finding that the majority of adults are unable to adhere to such a strict diet, and that managing PKU with diet alone does not lead to optimal patient outcomes.

MDDA’s position is that all Australians living with PKU should have access to all treatments that have been approved as safe and effective by the Therapeutic Goods Administration (TGA). This means that Australian clinicians should be given the choice of selecting the treatment that best meets the needs of each individual patient and will lead to optimal treatment outcomes – without any limitations or restrictions.

MDDA supports access to any new treatment that has been found to help manage this serious and rare condition, ensuring the brain health of those living with PKU is protected throughout life. Over the coming weeks and months, in the lead up to the PBAC’s July Meeting, MDDA will be strongly advocating for both Kuvan and Palynziq to be listed on the PBS for adults with PKU – and we need your help.

How you can help?

There are a number of ways you can help to support our advocacy efforts and ‘Make Some Noise for PKU’:

  1. Sign up to our ‘Make Some Noise for PKU’ campaign Facebook page, to stay informed of all advocacy activities and resources:
  2. PBAC Consumer Comments: You, your family members, friends and community can submit an online consumer comment to the PBAC, which will be considered when it meets to review Kuvan and Palynziq in July. Insights from patients and members of the community can provide valuable information to the PBAC about the impacts of living with PKU, and the potential benefits of the treatments being considered. Consumer comments will be open for eight weeks, from Wednesday 30 March-Wednesday 25 May 2022.
  3. Engage your local Federal Member of Parliament (MP): With a Federal Election due to be held by 21 May, this is an ideal time to engage your current local Federal MP, or the local political candidate/s in your electorate, to educate them about PKU and some of the challenges associated with dietary therapy alone. A separate announcement with guidance on the best way to engage your Federal MP will be available shortly.
  4. Share your story with your local media: Sharing your own, or your family’s, personal story of living with PKU will help to raise much needed awareness and understanding among the local community. A separate media briefing will be available to members who wish to participate in this activity.

Special MDDA Member Event

What:        PBAC Information Session and Q&A – with Jo Watson, Deputy Chair, PBAC

When:      Tuesday, 19 April 2022

Time:        7.30pm-8.30pm

Format:   Online/virtual meeting (details to come; event will only be for those who have pre-registered)

MDDA is pleased to announce that we have been able to secure a special PBAC Information Session and Q&A for members, with the Deputy Chair and Consumer Advocate of the PBAC, Jo Watson.

Jo has kindly agreed to take us all through how the PBAC process works and what information the PBAC finds helpful in the consumer comments they receive.

While the online consumer comments for the July PBAC meeting will open this week, we encourage you to hold off making a comment until after this session, to ensure you are able to include the information the PBAC wants to hear. There will still be five weeks left to submit your consumer comments after this event.

Details to register for this special event will be shared soon. Please note, you will need to register your attendance to participate in the event.

The power of advocacy

Our collective voices will be critical in ensuring our PKU community can access the latest medicines and treatments as they become available. We should not be left behind.

There is much work ahead in order to secure equitable treatment access for Australians living with PKU – but together, we can ‘make some noise for PKU’, by advocating for change and help to improve the lives of those living with this serious genetic metabolic disorder.

We look forward to keeping you updated on all developments with this advocacy campaign. Questions can be directed to [email protected] . Thank you for your support.


Monique Cooper                                                                                     Louise Healy

President                                                                                                        Vice-President