Healthcare Industry News: Mucopolysaccharidosis
News Release - January 25, 2006
BioMarin Receives FDA Fast Track Designation for Phenoptin for PKUNOVATO, Calif., Jan. 25 (HSMN NewsFeed) -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN News) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Phenoptin(TM) (sapropterin dihydrochloride) for phenylketonuria (PKU). Phenoptin, an investigational oral small molecule therapeutic, is currently in Phase 3 clinical development.
Pursuant to the Food and Drug Administration Modernization Act of 1997, FDA's Fast Track program 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.
"We are pleased that the FDA recognizes that PKU is a serious disease and unmet medical need, and we look forward to working closely with the agency in our effort to develop Phenoptin as the first drug therapy for its treatment," stated Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "In early December of 2005, we enrolled the last patient into the on-going Phase 3 clinical trial of Phenoptin, keeping us on-track to announce top-line results from the double-blind, placebo-controlled portion of this trial in late March 2006."
Phenoptin is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Phenoptin, sapropterin dihydrochloride, is the synthetic form of 6R-BH4, a naturally occurring enzyme cofactor. BioMarin received orphan drug designation for Phenoptin to treat PKU from both the FDA and European Medicines Agency. If Phenoptin is the first drug therapy approved for the treatment of PKU, BioMarin would receive seven years of market exclusivity in United States and 10 years in the European Union for this indication. BioMarin estimates that Phenoptin could be a potential treatment option for approximately 30 percent to 50 percent of the estimated 50,000 individuals diagnosed with PKU in the developed world.
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 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 global newborn screening efforts implemented in the 1960s and early 1970s, virtually all PKU patients 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 find difficult to maintain.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio is comprised of three approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(TM) (galsulfase) for Mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, AldurazymeŽ (laronidase) for Mucopolysaccharidosis I (MPS I), and OrapredŽ (prednisolone sodium phosphate oral solution) for inflammatory conditions. Investigational product candidates include Phenoptin(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (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: the development and commercialization of Phenoptin; and actions and timing of actions by regulatory and governmental authorities, particularly with respect to Phenoptin. 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 content and timing of decisions by the FDA and European Commission and other regulatory authorities concerning Phenoptin; the results of current and future clinical trials of Phenoptin; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Factors That May Affect Future Results" in BioMarin's 2004 Annual Report on Form 10-K and the factors contained in BioMarin's reports on Forms 10-Q and 8- K. In particular, fast track status does not guarantee a faster review time or approval for Phenoptin. 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.
NOTE: AldurazymeŽ is a registered trademark of BioMarin/Genzyme LLC.
OrapredŽ is a registered trademark of Medicis Pediatrics, Inc. and is used under license.
Source: BioMarin Pharmaceutical
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