BioMarin Receives Notice of Allowance for Once Daily Dosing Patent for Kuvan
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NOVATO, Calif., March 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical
Inc. (Nasdaq: BMRN) announced today that it has received a notice from the
United States Patent Office reporting allowance of claims covering once daily
dosing methods for Kuvan(R) (sapropterin dihydrochloride) in the treatment of
phenylketonuria (PKU). The company expects that the patent will be officially
issued later this year, and if issued, the patent's initial 20-year term would
expire in 2024. The company has a number of other pending patent applications
covering various aspects of Kuvan compositions and dosing.
"We believe the issuance of this patent will be significant in strengthening
our proprietary position on Kuvan," said Jean-Jacques Bienaime, Chief
Executive Officer of BioMarin. "This patent would prevent potential
competitors from using or copying Kuvan's approved dosing regimen. Based on
the claims allowed by the USPTO, we believe that the once daily dosing regimen
will prevent therapeutically equivalent generic competition to Kuvan while the
patent is in force."
About Kuvan
Kuvan (sapropterin dihydrochloride) Tablets are indicated in the United States
to reduce blood phenylalanine (Phe) levels in patients with
hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive
phenylketonuria (PKU). Kuvan is to be used in conjunction with a
Phe-restricted diet.
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.
Kuvan has received orphan drug designation from both the U.S. Food and Drug
Administration (FDA) and the European Medicines Agency (EMEA). Kuvan has
received seven years of orphan exclusivity in the United States and ten years
of market exclusivity in the E.U.
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 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. To learn more about PKU, please visit 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
three approved products and multiple clinical and pre-clinical product
candidates. Approved products include Naglazyme(R) (galsulfase) for
mucopolysaccharidosis VI (MPS VI), a product wholly developed and
commercialized by BioMarin; Aldurazyme(R) (laronidase) for
mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a
50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin
dihydrochloride) Tablets, a product for the treatment of phenylketonuria
(PKU), developed in partnership with Merck Serono, a division of Merck KGaA of
Darmstadt, Germany. Other product candidates include PEG-PAL (PEGylated
recombinant phenylalanine ammonia lyase), which is currently in Phase 1
clinical development for the treatment of PKU. For additional information,
please visit 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: expected action by the United States Patent and Trademark
Office, the continued development and commercialization of Kuvan and
BioMarin's other products and product candidates and actions by regulatory
authorities. 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: actions
by the United States Patent and Trademark Office, our success in the continued
commercialization of Kuvan; the content and timing of decisions by the U.S.
Food and Drug Administration, the European Commission and other regulatory
authorities concerning each of the described products and product candidates;
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 2008 Annual Report on Form
10-K, as amended, and the factors contained in BioMarin's reports on Form 10-Q
and 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.
BioMarin(R), Naglazyme(R) and Kuvan(R) are registered trademarks of BioMarin
Pharmaceutical Inc.
Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.
Contact:
Investors Media
Eugenia Shen Susan Berg
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 506-6570 (415) 506-6594
SOURCE BioMarin Pharmaceutical Inc.
Investors, Eugenia Shen, +1-415-506-6570, or Media, Susan Berg,
+1-415-506-6594, both of BioMarin Pharmaceutical Inc.
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