NHS England has today (17 December 2021) announced that it will make the medicine sapropterin for PKU available to patients of all ages. A deal has been struck for the unbranded generic version of Kuvan (sapropterin) which means the medicine will be available to all patients in England.
What is NSPKU’s statement on this news?
People with PKU and their families have worked hard with NSPKU to get to this announcement. PKU is a lifelong condition and we welcome the news that sapropterin will be available to all adults. We thank everyone that has contributed to NSPKU and our campaigns.
What is (sapropterin) Kuvan and what does it do?
Sapropterin is a medicine for PKU. The brand name for this medicine is KUVAN. Sometimes sapropterin is also called BH4.
We have a full factsheet here https://www.nspku.org/download/kuvan-facts/
How will I know if sapropterin will work for me?
Sapropterin does not work for all patients with PKU. This will be determined by a process of genetic testing (this is where a sample of your blood is analysed to check which PKU genetic mutations you have) and response testing (this is where you take sapropterin for a period of time to check the effect on your blood phenylalanine levels). More guidance on this process will be made available soon to help patients understand what will happen.
How soon will it take to start sapropterin response testing for adults?
We expect sapropterin to become available in January. However 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.
Do I need to contact my clinic about access to Kuvan?
NSPKU will seek more updates about the arrangements that will be made. We will make further announcements. We suggest you don’t contact your clinics separately at this time as this may put a strain on clinic staffing at a time which is already very challenging for the NHS.
I am not currently attending a metabolic clinic. What should I do?
If you are not currently registered with a metabolic clinic you should make contact with your clinic or GP. Your local GP will not be able to prescribe sapropterin but will be able to make a referral to a specialist metabolic clinic.
Guidance about different UK nations:
This new announcement currently only relates to England.
England: NICE guidance for using sapropterin in children and adults to the age of 22 is already in force. Sapropterin for adults age 22 and over will also become available under this new announcement.
Wales : NICE guidance for using sapropterin in children and adults to the age of 22 is already in force. Sapropterin for adults age 22 and over is not currently available under this new announcement. NSPKU will be in contact with the Welsh government about plans for extending access in Wales.
Northern Ireland : For children under 22 – 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. Sapropterin for adults age 22 and over is not currently available under this new announcement. NSPKU will be in contact with the NI government about plans for extending access in Northern Ireland.
Scotland : Currently sapropterin is not available in Scotland. NSPKU will be in contact with the Scottish government about plans for extending access in Scotland for patients of all ages.