FDA News

Biomarin Files New Drug Application with US FDA for its PEG-PAL Drug

(Nov 27, 2007)-- Biomarin Pharmaceutical announced that it has filed an investigational new drug application with the US Food and Drug Administration for its PEG-PAL drug, a treatment for a genetic disorder known as PKU.

The US company, which is listed in Switzerland, said it expects to initiate a clinical study of PEG-PAL (Poly Ethylene Glycol-Phenylalanine Ammonia Lyase) in patients with phenylketonuria (PKU) in the first quarter of 2008.

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, it said.

PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH), the company said.

PAH is required for the metabolism of phenylalanine, an essential amino acid found in most protein-containing foods.

If the PAH enzyme is not present in sufficient quantities, phenylalanine 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.

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