BioMarin Announces FDA Approval of Expanded Biologics Manufacturing Facility
NOVATO, Calif., Nov. 28, 2011 /PRNewswire/ — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received approval from the U.S. Food and Drug Administration (FDA) for the manufacturing facility expansion in Novato, CA. The facility will support up to $1.0 billion in revenue for BioMarin’s growing enzyme replacement therapy portfolio, which is comprised of complex glycoprotein products produced by mammalian cells.
“We are pleased with this first cycle approval of our manufacturing facility expansion which reflects our effective interactions with regulatory authorities and the high caliber of our technical operations team,” stated Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. “BioMarin has a strong track record in manufacturing enzyme replacement therapies. The approval of this facility supports the manufacturing requirements for anticipated peak sales of our commercial products and also supports several of our clinical and pre-clinical programs. BioMarin’s Novato manufacturing facilities are also producing clinical trial material for our pipeline protein products including, GALNS for MPS IV, PEG-PAL for PKU, our CNP analog for Achondroplasia and a new biologic to be disclosed at our upcoming R&D Day.”
Mr. Bienaime continued, “The facility expansion will also be the initial site of commercial production for GALNS for MPS IVA, if pivotal Phase 3 results are positive in the second half of 2012. The next phase in our manufacturing capabilities expansion will occur in our recently purchased facility in Ireland. Commercial production for some of our products in Ireland, including GALNS, will provide additional manufacturing diversity and assure adequate capacity for commercial demand of our current and future products.”
The multi-product manufacturing plant in Novato was first licensed for production of Aldurazyme for MPS I in 2003, then Naglazyme for MPS VI in 2005. This facility expansion, adjacent to the current commercial plant, is now approved to manufacture Naglazyme. This facility approval provides BioMarin not only additional capacity but also redundancy for its largest commercial product.
The scope of this project encompassed more than merely expansion of the existing facility. The scale of operations at the cell culture stage of production was doubled, the scale of operations at the initial purification stage of production was tripled, numerous technology and automation control enhancements were implemented to facilitate manufacturing flexibility in accordance with Good Manufacturing Practices, and High Temperature Short Time (HTST) technology for pre-treatment of media was implemented lowering the risk for potential viral contamination. The ability to gain rapid first cycle approval of this state-of-the-art manufacturing facility and associated process improvements is paramount to assuring our patients of uninterrupted supply of high quality product.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include NaglazymeÃ‚® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; AldurazymeÃ‚® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; KuvanÃ‚® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse(TM) (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, amifampridine phosphate (3,4-diaminopyridine phosphate), which is currently in Phase III clinical development for the treatment of LEMS in the U.S., PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, and BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers. 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 expectations of revenue and sales related to Naglazyme, Kuvan, Firdapse, and Aldurazyme; the financial performance of the BioMarin as a whole; the timing of BioMarin’s clinical trials of GALNS, Firdapse, PEG-PAL, BMN-673, BMN-701 and other product candidates; the continued clinical development and commercialization of Aldurazyme, Naglazyme, Kuvan, Firdapse, and its 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 Naglazyme, Kuvan, and Firdapse; Genzyme Corporation’s success in continuing the commercialization of Aldurazyme; results and timing of current and planned preclinical studies and clinical trials, particularly with respect to GALNS, Firdapse, PEG-PAL, BMN 673 and BMN 701; our ability to successfully manufacture our products and product candidates; 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; the market for each of these products and particularly Aldurazyme, Naglazyme, Kuvan and Firdapse; actual sales of Aldurazyme, Naglazyme Kuvan and Firdapse; Merck Serono’s activities related to Kuvan; 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 2010 Annual Report on Form 10-K, and the factors contained in BioMarin’s reports on Form 10-Q. 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.
BioMarinÃ‚®, NaglazymeÃ‚®, KuvanÃ‚® and Firdapse(TM) are registered trademarks of BioMarin Pharmaceutical Inc.
AldurazymeÃ‚® is a registered trademark of BioMarin/Genzyme LLC.
Contact: Investors Media Eugenia Shen Bob Purcell BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. (415) 506-6570 (415) 506-3267
SOURCE BioMarin Pharmaceutical Inc.