The NHS in Northern Ireland has decided to allow prescribing of sapropterin (previously known as Kuvan) for responsive patients with PKU of all ages
NSPKU is delighted to announce that the NHS in Northern Ireland is able to start routine prescribing of sapropterin for PKU for patients of all ages. This extends the previous policy which allowed prescribing for children and young people under the age of 22.
This announcement now means that all parts of the UK have equal access to sapropterin for patients of all ages. This was NSPKU’s campaign goal. NSPKU wishes to specificially thank Anne-Marie O’Loan and Chris Cassidy for their campaign and advocacy work in Northern Ireland over many years. NSPKU will be continuing to seek information about ensuring the NHS in Northern Ireland funds the clinic services necessary to support the roll-out.
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:
I have PKU and live in Northern Ireland. 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?
We understand that the details of the roll-out are not yet arranged. 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.
What is NSPKU view on new treatments for PKU?
NSPKU is delighted to hear that sapropterin is available to patients in Northern Ireland. NSPKU remains committed to seeking access to licensed and effective treatments for all patients with PKU in all parts of the UK. It is also essential that all clinic services, for all patients with PKU, are robust and adequately funded.