The Metabolic Dietary Disorders Association is extremely pleased with the decision of the Pharmaceutical Benefits Advisory Committee (PBAC) to recommend the listing of Kuvan (sapropterin dihydrochloride) on the Pharmaceutical Benefits Scheme (PBS) for maternal women living with phenylketonuria (PKU).
The PBAC noted there is a high clinical need in a small patient population, and acknowledged the input received from individuals, organisations and health professionals in relation to the November 2020 submission. Further, the PBAC noted the strong consumer feedback describing the very high clinical need for access to sapropterin for any adult with PKU. The PBAC would welcome a major resubmission for this broader population
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 PKU people who are responsive to this therapy. This welcome result has been the outcome of extensive ongoing political, media and public advocacy by our PKU and broader metabolic dietary disorders communities.
Whilst the outcome is a positive one for maternal women, we are pleased that the PBAC have noted the high clinical need for access to all adults with PKU. We intend to continue to campaign for access to all safe and approved treatments.
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 here.
There are still several processes to complete before Kuvan is available for those planning a pregnancy via your clinic. Once these processes are completed those with PKU who are planning a pregnancy should discuss responsiveness testing and other processes with their specialist metabolic clinic. MDDA will provide updates as they become available.