The UK MPS Society welcomes the news that the US Food and Drug Administration (FDA) has approved UX111 for the treatment of neurologic manifestations of Mucopolysaccharidosis Type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A, in paediatric patients with preserved neurodevelopmental function.
MPS IIIA is a rare, progressive and life-limiting lysosomal storage disorder that causes severe neurological decline and has a devastating impact on affected children and their families. MPS IIIA is increasingly recognised as a form of Childhood Dementia.
Today's decision by the FDA represents an important milestone for the global MPS community and reflects many years of research, clinical development and advocacy by families, clinicians, researchers and industry partners.
Sophie Thomas, Interim CEO of the UK MPS Society said:
This is a significant moment for the MPS IIIA community. Families affected by Sanfilippo syndrome have waited many years to see potential treatment options reach regulatory review, and today's decision will provide hope for many people around the world.
FDA approval applies only within the United States and does not automatically mean the therapy will be available in the United Kingdom.
While this approval is encouraging, the UK will need to undertake its own evaluation of this therapy before access can be considered here. UK evaluation will begin during mid-September 2026, with submissions to NICE expected approximately mid-November 2026 and the first NICE review meeting in mid-April 2027.
The MPS Society will continue to work with decision-makers, clinicians and the company to ensure that the needs of people living with MPS IIIA are fully understood throughout these processes.
