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News from
Biomarin.com, Nov. 27, 2007
BioMarin Files Investigational New Drug
Application for PEG-PAL for the Treatment of PKU
NOVATO, Calif., Nov. 27 /PRNewswire-FirstCall/
-- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced
today that it has filed an investigational new drug application
(IND) with the U.S. Food and Drug Administration (FDA) for PEG-PAL (Poly
Ethylene Glycol-Phenylalanine Ammonia Lyase), formerly known as
Phenylase(TM), for the treatment of phenylketonuria (PKU). The
company expects to initiate a clinical study of PEG-PAL in PKU
patients in the first quarter of 2008.
"Filing this IND is a notable milestone in the advancement of our
PEG-PAL program and our overall commitment to support the PKU
community," said Jean-Jacques Bienaime, Chief Executive Officer of
BioMarin. "With the Kuvan action date quickly approaching and the
completion of the PEG-PAL IND filing, we are continuing our progress
towards providing PKU patients with important new treatment options."
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. The patients in the Phase 1 study
will be offered continuation into a Phase 2 study that will evaluate
the safety and efficacy of weekly injections for eight weeks
followed by a dose titration period. Preclinical data has
demonstrated that PEG-PAL administered subcutaneously once weekly to
PKU mice resulted in a sustained decrease in blood phenylalanine (Phe)
levels in a twelve week study and has also shown potent Phe level
reductions in primates.
About PKU
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). PAH is required for the
metabolism of phenylalanine, an essential amino acid found in most
protein-containing foods. If the active enzyme is not present in
sufficient quantities, Phe 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. As
a result of newborn screening efforts implemented in the 1960s and
early 1970s, virtually all PKU patients under the age of 40 in
developed countries have been diagnosed at birth. Currently, PKU can
only be managed by a Phe-restricted diet, which is supplemented by
nutritional replacement products, like formulas and
specially-manufactured foods; however, the strict diet is difficult
for most patients to adhere to the extent needed for achieving
adequate control of blood Phe levels. To learn more about PKU,
please visit http://www.PKU.com. Information on this website is not
incorporated by reference into this press release.
About Kuvan
Kuvan(TM) is an investigational oral small molecule therapeutic for
the treatment of PKU. The active ingredient in Kuvan, sapropterin
dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin),
a naturally occurring enzyme cofactor that works in conjunction with
phenylalanine hydroxylase (PAH) to metabolize Phe. Clinical data
suggest that treatment with Kuvan results in significant reductions
in blood Phe levels in BH4-responsive patients. BioMarin and Merck
Serono estimate that Kuvan could be a potential treatment option for
approximately 30 percent to 50 percent of the estimated 50,000
identified PKU patients in the developed world.
Kuvan has received orphan drug designation from both the U.S. Food
and Drug Administration (FDA) and the European Medicines Agency (EMEA).
If approved, it will receive seven years of market exclusivity in
the United States and 10 years in the European Union for this
indication. Additionally, the FDA has granted Kuvan Fast Track
designation, which is designed to facilitate the development of new
drugs that are intended to treat serious or life-threatening
conditions and that demonstrate the potential to address unmet
medical needs.
About PEG-PAL
PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) is an
investigational enzyme substitution therapy for the treatment of PKU.
Pharmacology studies evaluated in the PKU mouse model demonstrated
that weekly subcutaneous administrations of PEG-PAL resulted in a
significant and stable decrease of plasma phenylalanine. BioMarin
estimates that PEG-PAL could be a potential treatment option for a
significant portion of the PKU population. BioMarin has filed an IND
application to the U.S. FDA and expects to initiate clinical testing
in PKU patients in 2008.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals
for serious diseases and medical conditions. The company's product
portfolio comprises two approved products and multiple clinical and
preclinical product candidates. Approved products include Naglazyme(R)
(galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly
developed and commercialized by BioMarin, and Aldurazyme(R) (laronidase)
for mucopolysaccharidosis I (MPS I), a product which BioMarin
developed through a 50/50 joint venture with Genzyme Corporation.
Investigational product candidates include Kuvan(TM) (sapropterin
dihydrochloride), a Phase 3 product candidate for the treatment of
phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications,
which is currently in Phase 2 clinical development for the treatment
of peripheral arterial disease and sickle cell disease. Both
products are being developed in partnership with Merck Serono, a
division of Merck KgA of Darmstadt, Germany. For additional
information, please visit http://www.BMRN.com. Information on
BioMarin's website is not incorporated by reference into this press
release.
Forward-Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: the development of its product
candidates PEG-PAL and Kuvan, and expectations regarding filings
with regulatory agencies. These forward-looking statements are
predictions and involve risks and uncertainties such that actual
results may differ materially from these statements. These risks and
uncertainties include, among others: the results of planned clinical
trials related to PEG-PAL; the content and timing of decisions by
the U.S. Food and Drug Administration, particularly with respect to
PEG-PAL and Kuvan, and those factors detailed in BioMarin's filings
with the Securities and Exchange Commission, including, without
limitation, the factors contained under the caption "Risk Factors"
in BioMarin's 2006 Annual Report on Form 10-K, as amended, and the
factors contained in BioMarin's reports on Form 8-K. Stockholders
are urged not to place undue reliance on forward-looking statements,
which speak only as of the date hereof. BioMarin is under no
obligation, and expressly disclaims any obligation to update or
alter any forward-looking statement, whether as a result of new
information, future events or otherwise.
SOURCE BioMarin Pharmaceutical Inc. - 11/27/2007
CONTACT:
Investors, Eugenia Shen, +1-415-506-6570, or
Media, Susan Berg, +1-415-506-6594,
both of BioMarin Pharmaceutical Inc.
Web site:
http://www.bmrn.com
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