| |
News from Biomarin August 13, 2007
Positive Kuvan(TM) Pivotal Phase 3 Trial Results Published in The
Lancet
NOVATO, Calif., Aug. 13 /PRNewswire-FirstCall/ -- BioMarin
Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that
final results from the Kuvan(TM) (sapropterin dihydrochloride)
pivotal Phase 3 clinical trial were published in the August 11,
2007, issue of The Lancet. The study suggests that treatment with
Kuvan results in significant reductions in blood Phe levels in some
phenylketonuria (PKU) patients. Kuvan, an investigational oral small
molecule for the treatment of PKU, is being developed in partnership
with Merck Serono, a division of Merck KGaA, Darmstadt, Germany.
"This marks the first published Kuvan clinical study, and we expect
additional publications in the coming months to build on the
collection of data related to Kuvan," said Emil Kakkis, M.D., Ph.D.,
Chief Medical Officer of BioMarin. "We recently received priority
review from the FDA and hope to receive FDA approval by late
November. In the interim, we are pleased to be able to provide Kuvan
to PKU patients prior to commercial availability through our
expanded access program."
The Phase 3 clinical study enrolled 89 patients with elevated blood
Phe levels aged eight years and above at 29 sites in the United
States, Europe and Canada. All patients enrolled had demonstrated a
reduction in blood Phe levels following treatment with Kuvan in a
Phase 2 screening study.
The patients were randomly assigned to receive placebo or 10 mg/kg
of Kuvan daily for six weeks. Patients were evaluated every two
weeks for changes in blood Phe levels and adverse events. The
primary objective was to assess the efficacy of Kuvan compared with
placebo for reduction of blood phenylalanine in patients with PKU.
The secondary objective was to assess the safety of Kuvan compared
with placebo. A total of 87 patients completed six weeks of
treatment.
Highlights from the Phase 3 double-blind study are summarized below:
Patients treated with Kuvan for six weeks had a mean decrease in
blood Phe level of 236 uM/L (29 percent) compared to a mean increase
of 3 uM/L (3 percent) in the placebo group (p<0.0001). Prior to
treatment, patients in the Kuvan group and placebo group had mean
blood Phe levels of 843 uM/L and 888 uM/L, respectively.
After six weeks of treatment, 54 percent of patients treated with
Kuvan and 23 percent of patients in the placebo group had a blood
phenylalanine concentration below the recommended 600 uM/L level
(p=0.003).
Blood phenylalanine concentrations fell by about 200 uM/L after one
week in the Kuvan group, and this reduction persisted for the
remaining five weeks of the study (p<0.0001).
The type and incidence of adverse events was similar in the Kuvan
and placebo groups. Kuvan was well tolerated and investigators
reported that no serious adverse event occurred.
About Kuvan
Kuvan 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. It also may enable
some patients to minimize or eliminate highly-restrictive dietary
constraints by increasing Phe tolerance levels. 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 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 (Phe), 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 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
candidate Kuvan; expectations regarding the Kuvan expanded access
program; expectations regarding filings with regulatory agencies;
and the development of 6R-BH4 for other indications. 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 ongoing clinical trials related to Kuvan; the
content and timing of decisions by the U.S. Food and Drug
Administration, the European Medicines Agency and other regulatory
authorities concerning Kuvan; results and timing of current and
planned clinical trials of 6R-BH4 for other indications; 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. - 08/13/2007
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
http://phx.corporate-ir.net/phoenix.zhtml?c=106657&p=irol-newsArticle&ID=1039688&highlight=
<----back |
|