Results of First Interim Efficacy Analysis for Riquent Phase 3 ASPEN Trial: Continuation of the Trial is Futile

BioMarin Pharmaceutical announced today that in the first interim efficacy analysis (IEA) for the Riquent(R) Phase 3 ASPEN trial, the Independent Data Monitoring Board (DMB) determined that the continuation of the trial is futile

NOVATO, CA, USA | February 12, 2009 | BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that in the first interim efficacy analysis (IEA) for the Riquent(R) Phase 3 ASPEN trial, the Independent Data Monitoring Board (DMB) determined that the continuation of the trial is futile. BioMarin and partner La Jolla Pharmaceutical have decided to stop the study, unblind the data and evaluate all of the clinical results including secondary endpoints such as SLE (Systemic Lupus Erythematosus) disease activity indices and proteinuria.

"While the results of this first interim efficacy analysis are obviously disappointing, we decided to take a calculated risk on this program and with the carefully staged agreement, we have spent a total of $15 million, a relatively modest amount for a late Phase 3 asset addressing a large market opportunity," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We remain dedicated to advancing our development pipeline by making investments in future growth and continue to look for attractive late-stage in-licensing or acquisition opportunities."

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three 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; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which was developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of sickle cell disease, and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 1 clinical development for the treatment of PKU. For additional information, please visit http://www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

SOURCE BioMarin Pharmaceutical Inc.

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