Treatment for PKU to be reviewed for reimbursement in Australia

Tuesday, 2 January 2018

Important Announcement for all Australian PKU Families

Please note the updated 2018 PBAC meeting schedule link including Kuvan consideration (pg20 in the document).

Important Announcement for MDDA Members and all Australian PKU Families

Treatment for PKU to be reviewed for reimbursement in Australia

Kuvan (sapropterin dihydrochloride) has been submitted to the Pharmaceutical Benefits Advisory Committee (PBAC) for consideration to be subsidised on the Pharmaceutical Benefits Scheme (PBS) for responsive patients with PKU. KUVAN is the first and only prescription medicine available for PKU.

MDDA is extremely supportive of this announcement as we have made several approaches to the makers of Kuvan, BioMarin, over the past 18 months in the hope that this submission would be made.

In some people with PKU, Kuvan is a drug therapy that, in combination with a low-protein diet, may help keep phenylalanine (Phe) levels in the blood lower. Kuvan may also help to control these Phe levels better than diet alone.

Elevated levels of Phe can be neurotoxic and impact the brain, potentially affecting the mood, energy and concentration of people with PKU. By reducing the Phe levels in people with PKU, Kuvan can help to minimise the impact of high Phe levels on the brain and potentially address these reversible neurotoxic effects.

MDDA feels that supporting new treatments that help us with the management of this serious condition is essential to ensure that the brain health of people with PKU is protected throughout life.

This is the second submission to the PBAC for Kuvan for the treatment of PKU – and there are no guarantees that it is going to be successful. Therefore, we need your help in ensuring appropriate consideration is given during the decision-making process, to support Kuvan’s listing on the PBS for Australians with PKU.

How you can help?

Over the coming weeks, we will be asking for your help in a variety of areas to make this campaign successful. There will be three key calls to action:

  1. Maximising consumer submissions to the PBAC to show the importance of listing Kuvan on the PBS for Australians with PKU.
  2. Generating awareness through social and mainstream media to get PKU in the spotlight in the lead up to the decision.
  3. Engaging with local MP’s to get their understanding and support for PKU and seek their help in advocating for our PKU community in Parliament.

Call to Action #1: PBAC Online Consumer Comments

Opens 20 December 2017 until 7 February 2018

If you are keen to support the availability of more treatments for PKU in Australia, it is important that you make a submission to the PBAC that will be considered as part of their evaluation of Kuvan. You could also ask friends and family to make a submission too.

Please look at the information below for guidance on how you can submit and online consumer comment for Kuvan.

Submissions should be in your own words, but you may want to consider the following:

  • How does living with PKU impact on you/your family member/friend?
  • Have you/your family member/friend ever experienced any health impacts from having PKU or high Phe levels, such as impact on mood or mental health?
  • What has been the impact on you/your family member/friend of not having access to new treatments for PKU?
  • What are the benefits of being able to access a treatment that could help reduce your Phe levels?

We will be in touch again soon with further information on other ways you can get involved to support this campaign.

Any questions can be directed to This email address is being protected from spambots. You need JavaScript enabled to view it..

Thank you,


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What is the Pharmaceutical Benefits Scheme (PBS)?
The PBS subsidises the cost of new medications needed by the Australian public. A PBS listing makes medications more affordable for the people who need them. Under the PBS, the Government covers most of the cost of a medication.

Who decides which medications are listed on the PBS? 
The Pharmaceutical Benefits Advisory Committee (PBAC) is an independent body that considers whether to add new medications to the PBS. The PBAC meets three times a year to review new medications for the PBS. It makes recommendations to Government about whether to list a medication on the PBS. The PBAC’s recommendations influence the Government’s decision-making on which medications to add to the PBS.

Who should comment on a PBS submission?
Anyone with an interest in a particular medication should submit a consumer comment, including people living with a disease, as well as anyone else impacted by the disease (i.e. family / friends / community members). 

The PBAC also welcomes comments from members of the public, patients, carers, healthcare professionals and patient or consumer groups. 

All comments received will be considered by the PBAC when it reviews a new medication.

The PBAC is interested in your personal experience (or on behalf of a group) on the potential benefits that a new medication will bring for patients, carers and their families. This may include, for example, the impact on quality of life and other benefits that will come about from a medication being added to the PBS or being reimbursed by the Government.

How to submit your comments on a PBS submission
Step 1: 
All medications being considered by the PBAC at its March 2018 meeting will be published on the Department of Health website ahead of time. The PBAC meeting agenda and a link to the consumer comment form is available on the PBS website ahead of every PBAC meeting.

The March 2018 meeting agenda will be published on 20 December 2017 at the following web address:

Consumer Comments for the March 2018 meeting can be submitted from:

20 December 2017 to 7 February 2018.

Step 2:  
There are three ways you can submit comments on a PBAC submission. Your comments must be submitted before the closing date (7 February 2018):

  1. By completing an online form on the Department of Health’s website. Available at the following address:   
  2. By requesting a printed form from the PBAC Secretariat (contactable on: 02 6289 7099) and returning the completed form to the Department of Health before 7 February 2018.
  3. By submitting a letter of support and sending it to the PBAC (address: GPO Box 9848, Canberra, ACT 2601) before 7 February 2018.

All comments submitted before the closing date will be considered by the PBAC.

When can patients or the general community find out if a PBAC application to fund a new medicine has been successful?

Whether the PBAC decides to recommend a new medication for introduction on the PBS or not, the outcome of its March 2018 meeting will be made publicly available six weeks after 9 March 2018, at the following address: 

Metabolic Dietary Disorders Association

This email address is being protected from spambots. You need JavaScript enabled to view it. 
03 9723 0600
Freecall: 1800 288 460
PO Box 33, Montrose VIC 3765