BioMarin Launches Firdapse in the European Union

NOVATO, Calif., April 19 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that Firdapse™ (3,4-diaminopyridine) is now commercially available in the European Union (E.U.) for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).  Launching immediately in Germany and the UK, the company expects to subsequently launch Firdapse in all major European markets by the end of 2010.  Firdapse received marketing approval in the E.U. for the treatment of LEMS in December 2009 and is the first approved treatment for this indication, thereby conferring orphan drug protection and providing ten years of market exclusivity in Europe.

"The launch of Firdapse brings the first specifically approved treatment option for LEMS to patients in the E.U. and marks our fourth commercial product on the market.  We look forward to meeting with the FDA in the second quarter of 2010 to determine the necessary regulatory path for Firdapse in the U.S., and we also continue to evaluate the best development strategy for this product in other indications in the U.S. and Europe," said Jean-Jacques Bienaime,  Chief Executive Officer of BioMarin.  "We remain committed to the advancement of our product pipeline and anticipate several notable upcoming milestones including results from the Phase I/II GALNS trial this current quarter and results from the Phase II PEG-PAL trial and Phase I trial of BMN-195 for Duchenne muscular dystrophy, both in the third quarter of 2010."

About LEMS

Lambert Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disease with the primary symptoms of muscle weakness.  Muscle weakness in LEMS is caused by autoantibodies to voltage gated calcium channels leading to a reduction in the amount of acetylcholine released from nerve terminals.  The prevalence of LEMS is estimated at four to ten per million, or approximately 2,000 to 5,000 patients in the EU and 1,200 to 3,100 patients in the U.S.  Approximately 50 percent of LEMS patients diagnosed have small cell lung cancer.

Patients with LEMS typically present with fatigue, muscle pain and stiffness.  The weakness is generally more marked in the proximal muscles particularly of the legs and trunk.  Other problems include reduced reflexes, drooping of the eyelids, facial weakness and problems with swallowing.  Patients often report a dry mouth, impotence, constipation and feelings of light headedness on standing.  On occasion these problems can be life threatening when the weakness involves respiratory muscles.  A diagnosis of LEMS is generally made on the basis of clinical symptoms, electromyographic testing and the presence of autoantibodies against voltage gated calcium channels.

About BioMarin

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 Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS).  Firdapse was originally developed by the pharmaceutical unit (AGEPS) of the Paris Public Hospital Authority (AP-HP) and is licensed from EUSA Pharma SAS.  Other product candidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU; GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase I/II clinical development for the treatment of MPS IVA and BMN 195, which is currently in Phase I clinical development for the treatment of Duchenne Muscular Dystrophy.  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 use of its product Firdapse; the potential market for Firdapse; expectations regarding filings with regulatory agencies; and the development of Firdapse 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 content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities, particularly the pending decision by the European Commission on the Marketing Authorization Application for such product, our success in the commercialization of such product, if approved; results and timing of current and planned preclinical studies and clinical trials related to such product; our ability to successfully manufacture the product; 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 2009 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.

BioMarin®, Naglazyme® and Kuvan® are registered trademarks of BioMarin Pharmaceutical Inc.

Firdapse™ is a trademark of BioMarin Huxley Ltd.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

SOURCE BioMarin Pharmaceutical Inc.