|
 |
| |
 |
|
|
|
|
6R-BH4
for Cardiovascular Indications
6R-BH4
(also referred to as tetrahydrobiopterin or BH4)
is a naturally occurring enzyme cofactor that is
required for numerous biochemical and physiologic
processes, including the synthesis of nitric oxide
(NO).
| 6R-BH4 at a Glance |
|
|
| • |
Several
Phase 2 and exploratory studies in the
BH4 cardiovascular program are underway
|
| • |
Data
from the Phase 2 peripheral arterial
disease (PAD) study is expected by late
2008 |
| • |
6R-BH4 is the same
enzyme cofactor used in Kuvan for the
treatment of PKU |
|
|
|
| |
|
|
| |
NO plays a key protective role throughout the
cardiovascular system. It has multiple positive
effects, such as relaxing smooth muscle, reducing
blood pressure, controlling inflammation and reducing
platelet aggregation. NO is synthesized in the
endothelium in a process that involves the enzyme
endothelial nitric oxide synthase (eNOS), the
substrate arginine, and the essential enzyme cofactor
BH4. A deficiency of BH4 can disrupt NO synthesis,
resulting in a loss of normal endothelial NO production.
This loss of endothelial NO production is commonly
referred to as endothelial dysfunction. Endothelial
dysfunction has been associated with many cardiovascular
diseases, including hypertension, diabetic vascular
disease, peripheral arterial disease, coronary
arterial disease and pulmonary hypertension, and
has been shown to be a strong predictor of cardiovascular
adverse events in a number of clinical studies.
In academic studies, researchers have demonstrated
that administration of BH4 improves vascular endothelial
function in animal models and in patients with
diabetes and other cardiovascular diseases. |
| |
|
|
| |
|
|
| |
BioMarin is evaluating the potential application
of 6R-BH4 in treating multiple cardiovascular
indications.
BioMarin is also performing a Phase 2 study in
sickle cell disease.
BioMarin has developed 6R-BH4 for the treatment
of phenylketonuria (PKU) and cardiovasular indications
in partnership with Serono. For additional information
about this partnership, please visiti the 'Business/Partnerships'
section of this website.
(For additional information about 6R-BH4 for
the treatment of phenylketonuria (PKU) please
visit the ‘Pipeline/PEG-PAL’ section
of this website.)
|
|
| |
|
|
| |
Back to Top |
|
| |
|
|
|
|
|
