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The UK Government: Approve Funding of Kuvan in the UK for the treatment of Phenylketonuria

This petition had 6,069 supporters

We are petitioning the British Government to make the drug Kuvan available on the NHS as a treatment for Phenylketonuria.

Imagine the fear and horror of finding out your new baby has an incurable rare disease that could cause brain damage? Imagine living your whole life not being able to eat most foods you buy in the supermarket because you have a rare disease? Imagine having to calculate, count and weigh every piece of food that goes into your mouth, every single day for your whole life. Imagine living life being different, all because you inherited a genetic mutation your parents had no idea even existed. Imagine spending your entire childhood being bullied because you can't eat what everyone else eats and you have to take a medicine that makes you feel sick and makes your breath stink, no matter how many times you brush your teeth. Now imagine there is a drug that could change your life, but you will never know how it feels to be normal because the government won't fund that drug and you can't afford to buy it yourself? We can tell you how that feels. Devastating. Soul crushing. We didn't choose to have a rare disease. Like around 1 in 10,000 babies born in the UK every year, we have Phenylketonuria (PKU).

There is a potentially life changing drug called Kuvan that could change this reality for people with PKU, but the British government is refusing to fund it. The PKU patients of the United Kingdom are fighting this as we believe their decision not to fund it is against our human rights.

Like all people with PKU, this diagnosis affects our whole lives, from birth until death. We have to follow an extremely restrictive low protein diet and drink a foul tasting, high volume medicine every day. Pregnant women with PKU are even more restricted, and are sometimes not able to consume any protein at all - and everything you can buy in the supermarket has at least a bit of protein in it. As PKU patients we can't eat most things other people take for granted. No meat, no fish, no dairy, no bread, biscuits, rice, pasta or soy. That means no cake or snacks at birthday parties, no pizza with friends, no chocolate, no ice cream, no quick trips out for a takeaway meal or a nice meal at a restaurant. No healthy, balanced meals. Imagine being a school child watching on while your classmates eat a treat that the teacher handed out? No weight loss diets, no Sunday roasts or quick, throw together meals. We eat nutritionally lacking, synthetic foods we can only get via prescription and some fruits and vegetables, and drink a foul tasting medicine that gives us synthetic protein, vitamins and minerals so we can function and survive.

PKU is an inborn error of metabolism where the body does not adequately break down Phenylalanine, a building block of protein, which then turn toxic in the bloodstream of a person with PKU. Kuvan is available in the United Kingdom which can increase a PKU person's protein tolerance, however the British government refuses to fund it on the NHS. The company that distributes Kuvan has offered every single PKU patient in the UK the opportunity to try Kuvan for free for one month on the grounds the NHS funds treatment thereafter, so no-one is even allowed to try it because the government says no. We are fighting to have this drug made available to all PKU patients - we believe that the denial of this medication by the British government is against our human rights. One parent in the UK has won the right for their child to take Kuvan on this basis and now we want it to be made available to all PKU patients in the United Kingdom. We need 100,000 signatures before the government will consider debating our petition in the House of Commons so please sign today and ask all of your friends and family to sign too.

People with PKU have to be on an extremely restrictive low protein diet for life in order to stop brain and neurological damage from high blood phenylalanine levels. While a normal person eats 60g-80g or more of protein per day, a person with PKU might eat as little as 2g of protein per day. To put this into perspective, one average slice of bread has between 4g and 5g of protein in it. Failure to adhere to the diet can result in severe brain damage and mental retardation, seizures, tremors, anxiety, depression, cognitive issues including speech, comprehension and other processing issues and many other frightening symptoms. On or off diet, there are significant psychological and psychosocial impacts on the PKU sufferer.

The British government refuses to fund Kuvan on the NHS, however the costs to purchase the drug out of pocket are not affordable for the vast majority of people. They argue that Kuvan is not a viable option as a medication as not everyone responds to it, and that those with especially low tolerances (called Classical PKU) will not respond at all. However, data coming out of the US suggests that this is not the case - while it is true that not all PKU patients are Kuvan responsive, in some cases patients with Classical PKU have tripled their tolerance after starting treatment with Kuvan. In addition to being very restrictive, the PKU diet is also expensive for and places great stress on both PKU patients and their families who may have to enforce the diet. Some adults find they cannot afford to remain on diet and so are forced to live life in a blur of the symptoms described above. For those who are responsive, Kuvan can help to change this.

Please sign our petition and give PKUers in the UK the chance to try Kuvan and to remain on it as a long term treatment option if they respond. The government happily funds expensive programs for illicit drug users, smokers and alcoholics who chose to risk their health. We did not choose to have a lifelong rare disease, but as a minority we are overlooked and it's time for that to change. Help us to fight for our human right to access this medication and have the chance to achieve a more normal life.

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