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 as a potential to treat patients whose blood Phe levels are not adequately controlled by 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.
