Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what progress NHS England and NICE have made on deciding whether to (a) license Kuvan and (b) make available Peg-Pal for the treatment of phenylketonuria.

Decisions on licensing are made by the relevant regulatory body, either the European Medicines Agency or the Medicines Healthcare products Regulatory Agency.

Kuvan (sapropterin) is already licensed for use in the treatment of phenylketonuria and the National Institute for Health and Care Excellence (NICE) is currently developing guidance on its use through its technology appraisal programme. NICE technology appraisal guidance makes recommendations for the National Health Service on whether drugs represent a clinically and cost-effective use of NHS resources, and NHS bodies are legally required to fund drugs recommended by NICE. The appraisal of Kuvan is formally due to begin the week commencing 15 October with final guidance anticipated in August 2019.

Peg-Pal (pegvaliase) does not yet have a licence for use in the treatment of phenylketonuria in the United Kingdom. NICE is currently considering whether pegvaliase, if licensed, would be suitable for a technology appraisal. If it is suitable and referred to NICE, NICE will aim to publish guidance on the use of pegvaliase as close as possible to licensing.

The Department has made no assessment of the cost per patient of not prescribing Kuvan or Peg-Pal. NICE will take all the health-related costs and benefits associated with treatment with the drugs into account in developing recommendations on whether the drugs represent a clinically and cost-effective use of NHS resources.