The Metabolic Dietary Disorders Association acknowledges the decision by the Pharmaceutical Benefits Advisory Committee (PBAC) to defer the listing of Palynziq (pegvaliase) on the Pharmaceutical Benefits Scheme (PBS) for Australians living with phenylketonuria (PKU).
Information regarding the PBAC outcome and a brief summary can be found here.
We know this outcome will be disappointing for many in our community who were hoping for a clear positive recommendation. A deferral is not a rejection, but it also does not mean that PBS listing is guaranteed. It indicates that the PBAC recognises the potential benefit of Palynziq for some people with PKU and requires further work before it can make a final decision.
The deferral allows the PBAC and the sponsor company, BioMarin, to explore what additional information, conditions or arrangements may be needed to progress consideration of Palynziq for PBS listing. This process can take time, and there is no certainty about the final outcome.
With the support of our PKU community, we have been actively advocating for many years to secure access to all safe and effective treatments for Australians with PKU who are responsive to therapy. This has included political engagement, media activity and public advocacy by people with PKU, families, clinicians and the broader metabolic dietary disorders community.
We were encouraged to see the PBAC acknowledge the input received from individuals, organisations and healthcare professionals in support of this submission. We sincerely thank everyone who shared their experiences and submitted consumer comments, your voices were heard and recognised.
We also want to thank each and every one of you who has contributed to this advocacy effort so far. We know the emotional toll that repeated submissions and uncertain outcomes can have, and we do not take your involvement for granted.
The Metabolic Dietary Disorders Association will continue to advocate with the Australian Government and other stakeholders for equitable access to safe and effective treatments for people living with PKU, including Palynziq. We will keep the community updated as we learn more about any next steps or developments, understanding that progress may be gradual and outcomes are not guaranteed.