Phenoptin™ (sapropterin dihydrochloride) is an investigational oral small molecule, being developed by Merck Serono (NYSE: SRA, VTX: SEO) in partnership with BioMarin Pharmaceutical (Nasdaq: BMRN), for the treatment of patients with phenylketonuria (PKU), who have elevated phenylalanine (Phe) levels.
A genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, PKU is caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH) – an enzyme required for the metabolism of phenylalanine (Phe), an essential amino acid found in most protein-containing foods.
Insufficient amount of PAH leads to accumulation of Phe to abnormally high levels in the blood and brain, which then results in a variety of complications: severe mental retardation and brain damage, mental illness, seizures and tremors, and cognitive problems.
The abovementioned Phase III diet study investigated Phenoptin™ (sapropterin dihydrochloride) combined with diet in 4-12 year old PKU patients.
The results show that all pre-specified efficacy and safety endpoints of the double-blind, placebo-controlled study were met. Phenoptin treatment caused a significant increase in phenylalanine tolerance as well as a reduction in blood phenylalanine levels. In addition, the data showed that Phenoptin was well tolerated in younger PKU patients who were under dietary control.Find more details of the clinical data from the BioMarin press release.
hello..im very happy to find this site. im a nuutritionist from iran. we have 800 patient in iran. rate of pku is very hight in my country.if i can realated with another person its my pleasure. thx.
Posted by: kazemi | February 7, 2008 12:29 AM | Permalink to Comment