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Kuvan Receives Marketing Approval in the European Union

December 9, 2008

NOVATO, Calif., Dec. 9 /PRNewswire-FirstCall/ — BioMarin Pharmaceutical
Inc. (Nasdaq: BMRN) announced today that its partner Merck Serono, a division
of Merck KGaA, Darmstadt, Germany, has received marketing approval for
Kuvan(R) (sapropterin dihydrochloride) for the treatment of
hyperphenylalaninemia (HPA) in phenylketonuria (PKU) or BH4 deficient patients
from the European Commission. Merck Serono estimates that there are
approximately 35,000 patients diagnosed with hyperphenylalaninemia due to PKU
or BH4 deficiency in the European Union. BioMarin will receive a milestone
payment of $30 million for this marketing approval along with royalties on net
sales of Kuvan in the E.U.

“Along with our partner Merck Serono, we are excited to bring the first
therapeutic option to manage PKU to the European market and expand the
geographic reach of Kuvan,” said Jean-Jacques Bienaime, Chief Executive
Officer of BioMarin. “We are focused on the continued expansion of Kuvan in
the U.S. and look forward to working closely with Merck Serono on the
successful launch of the product in the European market.”

About Kuvan

Kuvan(R) (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 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 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 6R-BH4 for cardiovascular
indications, which is currently in Phase 2 clinical development for the
treatment of peripheral arterial disease and sickle cell disease, and 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 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 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: 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 2007 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.


Source: newswire



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