Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.BioMarin Pharmaceutical has filed an investigational new drug application with the FDA for PEG-PAL, formerly known as Phenylase, for the treatment of phenylketonuria. The company expects to initiate a clinical study in the first quarter of 2008.
Subscribe to our email newsletter
The Phase I study will assess the safety and pharmacokinetics of single injections of PEG-PAL (poly ethylene glycol-phenylalanine ammonia lyase) in approximately 35 phenylketonuria (PKU) patients in a series of escalating dose cohorts. The patients in the Phase I study will be offered continuation into a Phase II study that will evaluate the safety and efficacy of weekly injections for eight weeks followed by a dose titration period.
The Phase I study will assess the safety and pharmacokinetics of single injections of PEG-PAL in approximately 35 PKU patients in a series of escalating dose cohorts. The patients in the Phase I study will be offered continuation into a Phase II study that will evaluate the safety and efficacy of weekly injections for eight weeks followed by a dose titration period.
Preclinical data has demonstrated that PEG-PAL administered subcutaneously once weekly to PKU mice resulted in a sustained decrease in blood phenylalanine (Phe) levels in a twelve week study and has also shown potent Phe level reductions in primates.
PKU is a genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH), which is required for the metabolism of phenylalanine, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures, tremors, and limited cognitive ability.