Following Wednesday’s announcement from Denali Therapeutics that the U.S. Food and Drug Administration has approved AVLAYAH, an enzyme replacement therapy for the neurological aspects of MPS II Hunter syndrome, we recognise that this news may raise many questions within our community.
This approval is an important and encouraging milestone and marks meaningful progress toward future therapies.
While this is a positive step in the United States, it does not mean the treatment is immediately available in the UK. A separate review process is required through UK regulators and funding bodies, including NICE. This process can take time, and we are not yet able to provide a clear timeline for access. We are actively engaging with Denali and will be involved in discussions as this progresses.
There are no immediate changes to current care. Patients should continue with their existing treatments as advised by their clinical teams.
MPS Society is actively involved in ongoing discussions with key stakeholders in the UK approval process. We will keep families informed and ensure that your voices are represented throughout.
If you have any questions or concerns, please contact our support team – we are here to help.
