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Kuvan's price as a miracle drug for people with PKU?

Updated on December 28, 2010

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BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) is a company that has been heavily involved in researching and development of a drug for individuals with a rare genetic disorder, Phenylketonura (PKU). PKU is a metabolic disorder in which individuals lack an enzyme to break down a specific amino acid Phenylalanine. The amino acid builds up in the body and can lead to various problems the worst being mental retardation in patients if left untreated. Currently the only form of treatment is dietary restriction of high protein foods and supplemented by a food supplement or formula. Kuvan is the first drug on the market to effectively treat this.

At the time of release the expected price tag for this drug is expected to be between $30,000 and $200,000 at a $.29 per milligram according to Kuvan statements. The biggest question is the insurance companies and how they will respond to this. Most insurance companies currently fight to stop individuals with PKU from getting the necessary food supplements they need in order to function. It will be interesting to see if the insurance companies will cover this, despite assurances from BioMarin. However a program that BioMarin has put in place to help it's customers called BPPS will help you get the needed drugs, and put you in contact with the National Organization of Rare Diseases (NORD). NORD has many programs that will help PKU patients, from paying for some foods and formula to covering the co-pay for Kuvan itself. (Mine is currently $400 per 3 months for co-pay.)

Many patients that are in the trials have varied results with Kuvan ranging some from nothing to upwards of a 70% effectiveness. This presumes they also still need to be on some form of dietary restriction. Typically you are instructed not to change your diet when you start the medication, but if it is effective then you will be able to change some of the food that you eat. Food Supplements or Formula which are an amino acid drink designed to replace the amino acids an individual would receive from higher protein foods are a struggle to get covered by insurance companies. Most insurance companies do not see these as a medical necessity and can cause headaches when it comes time for them to be covered. Eventually after a few weeks to months of back and forth they generally cover the foods supplement.

Perspective for Parents

It can be difficult to figure out if this is something you should have your children try. As a PKU adult and speaking from experience it depends on the age. When they're very young up until they hit the teens I would recommend keep them off it work the diet as they're supposed to. If you put them on when they're young they're growing and it will mean phe intake will change. They also might get a skewed view of their diet and believe they can eat certain things when they can't. This is especially important if they're over at a friends house and in the past they may have to tell someone they can't have xyz. However, if they're on Kuvan they may decide they can have it without even thinking when it could be the entire intake of Phe for a week.

When they become a teenager would probably be the best time to try Kuvan out. Even if it does not work for the PKU individual the side effects of better cognitive function and concentration amongst others that are positive will allow the teenager to make better choices. This will allow them to stabilize the levels, this is also the age they tend to cheat on the diet. I used to steal potato chips from the cupboard which would raise my levels too high. In social settings it can also make it even easier to deal with the food situation.

If you're an adult and not on Kuvan I highly suggest you try it out and read my personal update below.

Personal Update

I wrote the above article some years ago when Kuvan was first being brought onto the market. I think that there is much wider use than in the past. However, there are over 100+ genetic mutations for Phenylketonuria and as a result Kuvan doesn't repond to everyone. I've been taking Kuvan for about three years now and it has helped me greatly. I was fortunate to respond to Kuvan and it has lowered my blood levels from the high teens, to the mid single digits. I went from a level of 18 phe, to approximately 2, with in the first three week trial. I've now since leveled off at about 7 or so. If you have PKU get in touch with BioMarin, as they will give you a free 30 day trial of the product if you have a prescription from your doctor for it. I highly recommend it.

For more information visit or watch the video below.


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      3 years ago

      - For the longest time I had this pain in the tips of my finergs. It was like the nerves of my finergs were messed up. I couldn't grip anything because it felt like there were needles in my finergs. It was horrible. I decided to cut out diet coke completely. I did it and now the pain is gone. It would come and go but haven't had it since. Even splenda. The other day I started drinking more coffee. After a week my collar bone started hurting and my shoulder started to pop. That is the first step to the nerve problem. I cut out the splenda and both issues were gone. For some reason they affect me really badly. Thankfully I finally listened to my body and stopped drinking the poison.

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      3 years ago

      Boom shaaklaka boom boom, problem solved.

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      3 years ago

      How can the added value of a better qailuty of life for rare disease patients be measured? how can their active contribution to the society (their integration at school or at work), thanks to an appropiate treatment be assessed? How can the savings of hospital stay, because a good access to an existing orphan drug be evaluated?Governments spend a good deal of money to rescue a climber,, a fishing boat from the XXI century pirates, or to find bodies for the confort of the families, even when it was supposed to undergo a difficult financial situation Nobody questions these admirable actions, no matter how expensive they are, but everybody does when it comes tto the treatment of a person with a rare disease. Are they second row citizens?The economical crisis may prevent new patients from partipating in clinical trials or benefit from new treatments, that is why a survey on this matter is necessary to have concrete data. The inequalities of access to treatments can even be seen among regions or hospitals in the same country, when it depends on their budget or decision, as you mention.

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      4 years ago

      My Daughter is 24 and has PKU and is currently taking Kuvan. She has responded very well to it and yes, Biomarin has helped 100% with our insurance company!! It was difficult getting our Military health insurance to cover the formula and Kuvan .. but Biomarin had experience with Tricare and was able to get the coverage for us on their own. I will always be grateful for their involvement in the process!


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