July 16, 2008
Biopten (Sapropterin Dihydrochloride) Approved By Japanese Ministry of Health for the Treatment of PKU
NOVATO, Calif., July 16 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that Asubio Pharma Co., Ltd. (a subsidiary of Daiichi Sankyo), has received marketing approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) for a label extension of Biopten(R) (sapropterin dihydrochloride), which contains the same active ingredient as Kuvan(R) in the U.S., for the treatment of patients with phenylketonuria (PKU). BioMarin will receive a milestone payment of $1.5 million for this marketing approval along with double-digit royalties on net sales of Biopten for PKU in Japan under an exclusive license of data and intellectual property contained in the Kuvan new drug application.
"We have a long-standing relationship with Asubio as their early work on BH4 was instrumental to our success and accelerated timeline in the development and approval of Kuvan in the United States," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We are proud to work with Asubio to further expand our geographic reach and help to bring the first drug treatment option to PKU patients in Japan. We are also looking forward to the pending Kuvan approval by the EMEA, which we expect by the end of the year."
Kuvan(R) (sapropterin dihydrochloride) Tablets is indicated 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. 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). Kuvan has received seven years of market exclusivity in the United States. In November 2007, Merck Serono submitted a Marketing Authorization Application (MAA) to the EMEA for sapropterin dihydrochloride as an oral treatment for patients suffering from hyperphenylalaninemia (HPA) due to PKU or BH4 deficiency. If approved in the EU, it will receive 10 years of market exclusivity for this indication.
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.
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 preclinical 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, 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.
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), and Naglazyme(R) and Kuvan(R) are a 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
BioMarin Pharmaceutical Inc.
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/