NOVATO, Calif., Oct. 24 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. announced today that it has provided a $250,000 grant to the National Organization for Rare Disorders (NORD) to support a new financial assistance program for patients with phenylketonuria (PKU), a rare genetic metabolic disorder. Funds from this new program may be renewed based on ongoing need and are specifically directed to help PKU patients with the substantial daily cost of medical foods when not paid for by insurance or Medicaid programs. NORD is working closely with Genetic Metabolic Dietitians International (GMDI), an independent professional organization, to develop program guidelines and provide ongoing support to NORD in the administration of these funds.
“We are committed to the PKU community and believe that it is important that every patient obtains optimal therapeutic treatment,” said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. “We are pleased to expand our relationship with NORD and GMDI to assist patients with out of pocket costs and help to ensure that patients in need are not limited by financial constraints.”
“PKU patients must maintain strict diets and rely on costly medical foods which are not covered by most insurance companies. This can cost families thousands of dollars per year,” said Abbey Meyers, President and co-founder of NORD. “This grant will enable us to enhance our services to better meet the immediate needs of PKU patients and alleviate the considerable financial burden this illness has on their families.”
“Consistent with our mission to maximize the benefits of newborn screening and prevent the devastating outcomes of an untreated disease through the use of medical foods, we are pleased to work with NORD and BioMarin to help the PKU community. Together, we are dedicated to helping PKU patients manage the disease through a variety of offerings, including financial assistance for medical foods,” said Kathleen Huntington, MS, RD, LD, Metabolic Dietitian at the Oregon Health Sciences University and a board member of GMDI.
Additionally, BioMarin plans to provide support to PKU patients through its BioMarin Patient and Physician Support (BPPS) program. BPPS currently provides support for MPS VI patients treated with Naglazyme, and a similar support program will be available for PKU patients if Kuvan is approved.
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). 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 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. Currently, PKU can only be managed by a Phe- restricted diet, which is supplemented by nutritional replacement products, like formulas and specially-manufactured foods; however, the strict diet is difficult for most patients to adhere to the extent needed for achieving adequate control of blood Phe levels. 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 Kuvan
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 suggest that treatment with Kuvan results in significant reductions in blood Phe levels in BH4-responsive patients. It also may enable some patients to minimize or eliminate highly-restrictive dietary constraints by increasing Phe tolerance levels. 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). If approved, 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 of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
About BioMarin
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. Both products are being developed in partnership with Merck Serono, a division of Merck KgA of Darmstadt, Germany. For additional information, please visit http://www.BMRN.com. Information on BioMarin’s website is not incorporated by reference into this press release.
About NORD
The National Organization for Rare Disorders (NORD) is a unique federation of voluntary health organizations dedicated to helping people with rare “orphan” diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment and cure of rare disorders through programs of education, advocacy, research and service. NORD was established in 1983 by patients and families who worked together to get the Orphan Drug Act passed.
About GMDI
The mission of GMDI is to provide standards of excellence and leadership in nutrition therapy for genetic metabolic disorders through clinical practice, education, advocacy, and research. GMDI members are leading practitioners in the field of genetic metabolic nutrition who are joining together to identify education and practice needs, and develop standards of practice and training opportunities in this highly specialized field. Membership provides opportunities for professional education and research, as well as networking and peer support.
Forward-Looking Statement
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: expectations regarding Kuvan; the continued clinical development of 6R-BH4; and actions by regulatory authorities, including actions related to Kuvan and 6R-BH4. 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: results and timing of current and planned preclinical studies and clinical trials of 6R-BH4 for cardiovascular indications; actions related to 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 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 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.
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/
http://www.PKU.com/
