First Drug to Treat PKU Approved

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.

FDA Patient Safety News: March 2008

For more information, please see our website:
http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/psn/transcript.cfm?show=73#1

Category:

Education

Tags:

• Phenylketonuria
• PKU
• Kuvan
• FDA
• drug

[CrystalRocksThis]

no the disorder doesnt hurt us( if our diet is properly maintained) and the drug doesnt. it helps with the breakdown of phenylalanine :)

[SexyJamiesGirl]

does it hurt them

[gabby6741]

thank you, your video saved me..i have a project due about PKU and i couln't find any imformation about it

[NastynasThat]

they havnt cured it man i was on this bullshit 2 times.... didnt work

[goatmilk9]

I have PKU as well and I am just going to contiue taking my regular formula.

[17Blueyes]

I have P.K.U and I have taken it.unfortunately I was a non-responder...but I am continuing to take the formula...and til this day its still nasty to me now 26...joshua miller

[Cfranki001]

this drug will prob be super expensive and prob not covered. Your dieticians say its only for people with mild PKU? well wouldnt work for me. I have classical PKU. And learned to live with it. So, If they find a cure, cool! But I really don't find a diet that hard to stick with. Its just food we have to watch.. not like we have to inject ourselves with stuff or spend months getting traetments in the hospital right? good thing they figured out a diet plan that works.

[johnjmack]

Why no fair balance information?

[IchigoDancerNyan]

Nope, they didn't cure PKU.

They just made it to where we can take this and it makes us able to have a higher phe intake. It's so expensive though X_X I think they said it was $1000 per pound!!!

I went to a PKU clinic in california and they talked about this. They used a lot of big words XD

[driftking4]

i took this b4, didnt work for me... that sux