NHS England interim decision on sapropterin (Kuvan) for PKU

In July 2018, NHS England decided to develop an interim policy for the use of sapropterin for PKU. The draft policy recommended the use of Kuvan in children and adults of all ages who are responsive to the treatment. We have been informed that following the prioritisation process that took place in November, NHS England will not be funding the treatment for cost reasons.

NHS England’s usual process is to reconsider policies that were not funded in the next prioritisation round, which will take place in May 2019. NHS England has informed us that this issue is under consideration. The NSPKU will be pressing NHS England to reconsider its funding decision in the next round. 

NICE is also developing guidance on the use of Kuvan. This is a separate process, which is not affected by the decision taken by NHS England.

NHS England’s statement is as follows:

“NHS England has carefully reviewed the evidence to treat phenylketonuria with sapropterin. NHS England recognises that the published evidence identifies that, at present, there is sufficient evidence to commission this treatment. However, following the relative prioritisation process undertaken in November 2018 for funding interventions in 2019/20, NHS England has concluded that, balanced against other relative priorities that were also considered during this process, sapropterin to treat phenylketonuria will not be funded at this time within the resources available.”


The NSPKU welcomes that NHS England has finally recognised that Kuvan is an effective treatment with sufficient evidence to commission the treatment. Kuvan has been used in Europe for 10 years and is a standard treatment in almost every country in the developed world. It is very regrettable that NHS England has not funded the treatment at this time and we recognise this will be very disappointing news for many people living with PKU.

The NSPKU will be making a complaint about the poor planning and timing of this announcement immediately before Christmas, and will be urging NHS England to improve the way it communicates with rare disease charities.

The NSPKU is committed to pressing BioMarin and the NHS for a solution allowing for wide access to this treatment in all parts of the UK. NSPKU’s policy is to support the use of all safe and effective treatments to help people with PKU manage their condition. 

NSPKU helpline: 030 3040 1090

21st December 2018