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?
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. Submissions open 20 December to 7 February.
Please look at this information https://www.mdda.org.au/help/submit-a-pbs-consumer-comment/ 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 firstname.lastname@example.org.