First Drug to Treat PKU Approved (March 2008)

FDA has approved the first drug to treat phenylketonuria (PKU), which is a genetic disorder that affects one in about 15 thousand births in the U.S. The drug, called Kuvan (sapropterin dihydrochloride) was developed by BioMarin Pharmaceutical in partnership with Merck.

Those with PKU cannot metabolize phenylalanine, which is an amino acid found naturally in many foods. Normally, phenylalanine metabolism is controlled by the enzyme phenylalanine hydroxylase. In PKU patients, this enzyme does not function properly. That causes high blood levels of phenylalanine, which can lead to mental retardation, behavioral abnormalities, seizures and other neurological complications.

Kuvan increases the activity of phenylalanine hydroxylase in those PKU patients who have some residual functioning of this enzyme. This leads to improved breakdown and lower blood levels of phenylalanine. Still, in order for the drug to be most effective, patients on Kuvan must be on a phenylalanine-restricted diet, and blood levels must be monitored frequently.

The safety and efficacy of Kuvan was demonstrated in four short-term studies of 579 PKU patients. In addition, the manufacturer will establish general disease registries for PKU to help track clinical results and monitor any adverse effects.

Additional Information:

FDA Press Release. FDA Approves Kuvan for Treatment of Phenylketonuria (PKU). December 13, 2007.
http://www.fda.gov/bbs/topics/NEWS/2007/NEW01761.html

Category:

News & Politics

Tags:

• PKU
• Drug
• Approved
• Kuvan
• Sapropterin
• Dihydrochloride
• FDA

[jannabobable]

Nice video! There's a similar video on youtube that shows what happens if PKUs are NOT treated called "PK-who"

[a5c11char5et]

@reddragon585

I have PKU - as far as I am aware this drug is only really effective if the patient can metabolise more phe than is possible for many patients of classic PKU

[reddragon585]

Hmm... Im skeptical... Oh i wanna join in now! My husbands got pku. I dont... Im sorry for all of u, but atleast u dont have to partake in fattening fads like mcd's or hormone injected foods.

[yllib25]

yea ..well it does help stop retardation

retardation can only happen when your real young right???

[mmmkkk987]

i honestly don't see the point of this stupid drug, if you take it you still have to be on pku diet, so what's the point? The diet should keep your levels down, not this drug. They just waste time and energy trying to make this stuff which is supposedly going to help me and others with PKU, but they aren't doing anyhting for us because PKu isn't a fatal condition. I have learned to live with PKU, but it still bothers me sometimes and i'm 16

[yllib25]

yea me to bro only im US

this drug is a load of crap my docter was telling me how worthless it is, theres little chances it works for some.. patinents..

and the funny thing is if it was lucky enough to work for you it only lets u get like 1 ounce per so many pills. never waste ur money on kuvan

[koolkathansy]

i have pku im 15 from UK