|
 |
| |
 |
|
|
|
|
 |
PEG-PAL for
Severe PKU
PEG-PAL (PEGylated recombinant
phenylalanine ammonia lyase or ‘PAL’)
is an investigational enzyme substitution therapy
for the treatment of severe phenylketonuria (PKU),
an inherited metabolic disease caused by a deficiency
of the enzyme phenylalanine hydroxylase (PAH).
| PEG-PAL
at a Glance |
|
|
| • |
BioMarin filed an
IND for PEG-PAL in 2007 |
| • |
PEG-PAL has received
orphan drug status in the United States |
| • |
Soon, PEG-PAL will
be 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.
|
|
| |
|
|
| |
|
|
| |
The PEG-PAL Phase I trial is expected to begin
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 tremendous potential to treat the entire spectrum
of PKU patients by bringing their Phe levels down
to normal levels. |
|
| |
|
|
| |
|
|
| |
In late 2007, BioMarin filed an IND for PEG-PAL
and has received orphan drug status in the United
States. |
|
| |
|
|
| |
Back to Top |
|
| |
|
|
|
