PBAC Kuvan Deferral for maternal women with PKU

The Metabolic Dietary Disorders Association recognises the decision by the Pharmaceutical Benefits Advisory Committee (PBAC) to defer the listing of Kuvan (sapropterin hydrochloride) on the Pharmaceutical Benefits Scheme (PBS) for maternal women living with phenylketonuria (PKU). We understand that the PKU community may feel disappointed by this decision, however, the deferral means the PBAC recognises the benefit of Kuvan for maternal 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 for maternal PKU women.
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 Kuvan. Although the outcome has not been a straightforward recommendation, MDDA remain hopeful to secure subsidised access to Kuvan through the PBS for PKU women who are planning a pregnancy. We intend to continue to campaign for access to all safe and approved treatments for all Australians living with PKU. MDDA plan to investigate next steps of access to a generic option and other therapies currently available overseas.
We want to thank everyone who submitted a consumer comment to the PBAC about how important it is for PKU maternal women and our wider PKU community to get access to this treatment on the PBS.
Information regarding the PBAC outcome and brief summary can be found https://bit.ly/2Wt1oPe. MDDA will provide updates as they become available.
Any questions can be emailed to [email protected].