NSPKU Statement on Pegvaliase and Kuvan

Pegvaliase is an investigational enzyme substitution therapy for the treatment of PKU, which has been developed by BioMarin Pharmaceutical. It has recently been approved for the treatment of adults with PKU in the USA. The product needs to be reviewed and licensed by the European Medicines Agency (EMA) before it can be used in Europe (including the UK). We do not currently know the time frame of this procedure.

If Pegvaliase is licensed by the EMA, it will not automatically be an approved treatment for PKU for use by the NHS. The NHS typically conducts an appraisal of the benefit provided by a drug and its cost effectiveness. The NSPKU has recently submitted evidence to a scoping consultation by the National Institute for Health and Care Excellence (NICE), which is considering whether to appraise Pegvaliase.

The NSPKU is also engaging with the Scottish Medicines Consortium which is conducting an appraisal of Kuvan and expect to participate in a formal appraisal of Kuvan by NICE or NHS England later this year.

The NSPKU supports and strongly advocates for the availability of all safe and effective treatments for PKU. In all our engagements with the NHS or healthcare bodies we advocate for the widest possible access to treatment to benefit our patient community. The evidence we have gathered from the NSPKU survey is used to inform the NHS of the difficulties and challenges that many people with PKU experience.

In addition, the NSPKU is concerned that the drug appraisal systems of the NHS in the UK are unfair to rare diseases like PKU. Our political work across the UK aims to ensure the PKU community is not left behind by unfair drug appraisal rules. We also advocate for fair pricing by drug companies to aid access to treatment.

The NSPKU is doing everything it can to represent the PKU community. You can help us in our work by joining as a member, fund raising for research and advocacy and taking part in our political campaign.