PEG-PAL for PKU
PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase or ‘PAL’) is an investigational enzyme substitution therapy for the treatment of phenylketonuria (PKU), an inherited metabolic disease caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). PEG-PAL is being developed for patients who do not respond to Kuvan and/or for those who wish to reduce blood Phe levels beyond what is possible with Kuvan.
PEG-PAL at a Glance
- BioMarin submitted an IND for PEG-PAL in 2007
- PEG-PAL has been assigned the Orphan Drug designation in the United States
- PEG-PAL is now being evaluated in the clinic
Nearly all developed countries test for PKU as part of their newborn screening program. An estimated 50,000 people under 40 years of age in developed countries are living with the disease.
Preclinical Studies of PEG-PAL
The PEG-PAL Phase I trial was initiated in the first half of 2008 and it is hoped that positive preclinical data showing sustained decreases in blood Phe levels in PKU mice will be replicated in humans. If proven safe and effective, PEG-PAL has the potential to treat the entire spectrum of PKU patients by bringing their Phe levels down to normal levels.
