The team at the NCIMD (National Centre for Inherited Metabolic Disorders) has learnt that the NCPE (National Centre for Pharmacoeconomics) has recommended against the funding of Kuvan (sapropterin dihydrochloride) for the treatment of PKU. The NCIMD team is disappointed to learn of this decision. The team is aware that this treatment may have a beneficial effect for a small number of our patients in allowing some liberalisation of their diet. The team is also aware of the concern and anxiety that this decision has brought to our patients and families. However dietary treatment has been and remains beneficial and successful for over 60 years. The NCIMD will continue our support for the provision of Kuvan for the subgroup of patients who may benefit from it.
Dr. A.A. Monavari
Consultant Metabolic Paediatrician
Clinical Director, National Centre for Inherited Metabolic Disorders
Temple Street Children’s University Hospital