BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Kuvan(TM) (sapropterin dihydrochloride), an investigational oral small molecule for the treatment of patients with phenylketonuria (PKU) being developed in partnership with Merck Serono. Merck Serono expects to file a Marketing Authorization Application (MAA) for Kuvan with the European Medicines Agency (EMEA) in the third quarter of 2007.
"This NDA filing represents a significant milestone in our PKU program and our efforts to bring the first treatment option to PKU patients," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "The NDA filing contains data evaluating Kuvan in approximately 650 human subjects in six clinical studies and represents BioMarin's largest and most comprehensive filing to date. The company is now gearing up for the U.S. launch of Kuvan in late 2007 or early 2008."
At a pre-NDA meeting in October 2006, BioMarin met with the FDA to discuss key information on Kuvan to be included in the NDA. Consistent with the discussions at that meeting, the fully electronic NDA filing includes a comprehensive set of preclinical, clinical and manufacturing related data on Kuvan. As part of the NDA submission, BioMarin has requested priority review status. The criteria for assigning priority review status to an application are similar to those for fast track status, which Kuvan has already received. If the FDA accepts the NDA and grants the request for priority review, the FDA is expected to take action on the application within six months of its submission. Kuvan also has orphan drug designation, which allows for seven years of market exclusivity within the United States following the approval of the marketing application.
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 suggests that Kuvan produces 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). Upon approval, 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 and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
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 brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures and tremors, and cognitive problems. 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. The only treatment currently available for PKU patients is a highly restrictive and expensive medical food diet that most patients fail to adhere to the extent needed for achieving adequate control of blood Phe levels. To learn more about PKU, please visit 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 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. For additional information, please visit 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 development of its product candidate Kuvan; 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.
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