Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps his Department is taking to improve patient access to new and emerging treatments for Phenylketonuria, including those available in other countries.

The Government recognises how important it is that patients with rare diseases can benefit from access to effective new medicines. The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether new licensed medicines should be routinely funded by the NHS based on an assessment of clinical and cost effectiveness. NICE aims wherever possible to issue guidance on new medicines close to the time of licensing and the NHS is legally required to fund medicines recommended by NICE, normally within three months of the publication of final guidance.

NICE has published guidance recommending sapropterin (brand name Kuvan) for phenylketonuria (PKU) on the NHS as a possible treatment for people with this condition who meet the access criteria, are aged under 22 years old or who are pregnant.

NICE is also in the very early stages of developing technology appraisal guidance on sepiapterin for treating hyperphenylalaninaemia in phenylketonuria in people of any age. The evaluation is currently expected to begin in 2027.