NHS Scotland allows prescribing of sapropterin (previously known as Kuvan) for PKU

 NSPKU is delighted to announce that NHS Scotland is able to start routine prescribing of sapropterin for PKU.

We are informed that in Scotland sapropterin will be prescribed in line with the best practice UK consensus pathway from BIMDG.  It will be available to patients of all ages with PKU.

What is sapropterin (or Kuvan) and what does it do?

 Kuvan is the brand name for a medicine called sapropterin, for PKU.  Sapropterin is now available as an unbranded generic medicine – it is the same medicine as Kuvan but is made by a different company.

We have a full factsheet here:

https://www.nspku.org/download/kuvan-facts/

 I have PKU and live in Scotland.  Will I be able to take sapropterin?

Sapropterin does not work for all patients with PKU.  It depends which  PKU gene mutations the patient has.  It is estimated about 20-30% of patients in the UK will be responsive to sapropterin treatment.  Your clinic will need to find out whether you are responsive to sapropterin.  Your clinic will discuss this process with you.

How soon will it take to start sapropterin response testing?

 It is unlikely that clinics will be able to response test everyone straightaway.  It is important that the process is done carefully with supervision from your metabolic team.  You may need to wait to allow time for the clinics to do the response testing.  We suggest you don’t contact your clinics separately at this time as this may put a strain on clinic staffing.

Guidance about different UK nations:

England and Wales : Sapropterin is available.

Northern Ireland : The Department of Health, Social Services and Public Safety (DHSSPS) in Northern Ireland agreed to link to NICE. This means that they look at any guidance issued by NICE and decide if it is relevant for Northern Ireland. The DHSSPS usually approves most NICE guidance.

What is NSPKU view on new treatments for PKU?

NSPKU is delighted to hear that sapropterin is available to patients in Scotland.  NSPKU remains committed to seeking access to licensed and effective treatments for all patients with PKU in all parts of the UK.

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