MDDA welcomes the decision by the Pharmaceutical Benefits Advisory Committee (PBAC) to defer the listing of Kuvan (sapropterin hydrochloride) on the Pharmaceutical Benefits Scheme (PBS) for Australians living with phenylketonuria (PKU). Whilst it is disappointing not to have received a positive recommendation, the deferral means the PBAC recognises the benefit of Kuvan for some PKU patients and remains open to listing the treatment on the PBS.

The deferral also ensures the PBAC can now work with the sponsor company (BioMarin) to seek further evidence and determine the best pathway forward to make Kuvan available on the PBS.

MDDA, with the support of our PKU community, has been actively campaigning for a number of years to secure access to this treatment for all Australians with PKU who are responsive to therapy. This has included extensive political, media and public advocacy by our PKU and broader metabolic dietary disorders communities. Although the outcome has not been a straightforward recommendation, there is still hope to secure subsidised access to Kuvan – which is the first and only treatment available for PKU – through the PBS for some patients.

We were heartened to see the PBAC acknowledge the input received from individuals, organisations and healthcare professionals in support of this listing. We would like to thank everyone who submitted a consumer comment to the PBAC about how important it was for our PKU community to get access to this treatment on the PBS.

We were blown away to be told by the PBAC that it received 965 consumer comments in support of listing Kuvan on the PBS! This is not just extraordinary for a rare disease – but it is the first time the PBAC has ever received this volume of consumer comments for any treatment it has previously reviewed. This is a testament to the passion and commitment of our community and supporters and we can’t thank you enough.

We also want to thank each and every one of our supporters that helped to achieve this outcome. Whether it was raising the issue with your Federal Member of Parliament, sharing your story of living with PKU with media, or talking about the issue with your family and friends to raise awareness of PKU, this has been an enormous effort and we are very grateful and proud of the role everyone has played.

We will continue to work hard to advocate on behalf of Australians with PKU for access to available treatments with the Australian Government. We look forward to keeping you updated of any developments, as we keep pushing for reimbursement of Kuvan on the PBS.…/pbac-…/2018-03/deferrals-03-2018.pdf