Profile: Biomarin Pharmaceutical Inc. (BMRN.O)

BioMarin Pharmaceutical Inc. (BioMarin) develops and commercializes biopharmaceuticals for serious diseases and medical conditions. Its product portfolio comprises three approved products and multiple investigational product candidates. Approved products include Naglazyme (galsulfase), Kuvan (sapropterin dihydrochloride) and Aldurazyme (laronidase). As on December 31, 2008, BioMarin was conducting preclinical development of several other enzyme product candidates for genetic and other diseases, including a small molecule for the treatment of Duchenne muscular dystrophy. In February 2009, BioMarin announced the results from its Phase II multi-center, randomized, double-blind, placebo-controlled clinical study of 6R-BH4 in patients with symptomatic peripheral arterial disease (PAD). In October 2009, Rodman & Renshaw Capital Group Inc. announced that its affiliate, Aceras BioMedical LLC, has sold Huxley Pharmaceuticals Inc. to BioMarin Pharmaceutical, Inc.

On January 6, 2009, the Company announced its agreement with La Jolla Pharmaceutical Company (La Jolla), to develop and commercialize Riquent. On December 9, 2008, BioMarin announced its partner Merck Serono, a division of Merck KGaA (Darmstadt, Germany), received marketing approval for Kuvan for the treatment of hyperphenylalaninemia (HPA) in phenylketonuria (PKU) or BH4 deficient patients from the European Commission (EC). On October 15, 2008, BioMarin announced results from its Phase II multi-center, open-label, dose-escalation clinical study of 6R-BH4 in patients with sickle cell disease designed to evaluate whether 6R-BH4 can improve the endothelial dysfunction observed in sickle cell disease patients.

Commercial Products

Naglazyme is a recombinant form of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) indicated for patients with mucopolysaccharidosis VI (MPS VI). Naglazyme was granted marketing approval in the United States in May 2005 and in the European Union in January 2006. Naglazyme has been granted orphan drug status in the United States and the European Union, which confers seven years of market exclusivity in the United States and 10 years of market exclusivity in the European Union for the treatment of MPS VI, expiring in 2012 and 2016, respectively. BioMarin markets Naglazyme in the United States, European Union, Latin America and Turkey using its own sales force and commercial organization.

Kuvan

Kuvan was granted marketing approval for the treatment of PKU in the United States in December 2007. BioMarin markets Kuvan in the United States. Kuvan has been granted orphan drug status in the United States, which provides for seven years of market exclusivity in the United States for the treatment of PKU. Kuvan is a synthetic oral form of 6R-BH4, an enzyme co-factor for phenylalanine hydroxylase (PAH) indicated for patients with PKU.

Aldurazyme

Aldurazyme has been approved for marketing in the United States, European Union and other countries for patients with mucopolysaccharidosis I (MPS I). Aldurazyme has been granted orphan drug status in the United States and the European Union, which gives Aldurazyme seven years of market exclusivity in the United States and 10 years of market exclusivity in the European Union for the treatment of MPS I, expiring in 2010 and 2013, respectively. The Company has developed Aldurazyme through a 50/50 joint venture with Genzyme Corporation.

Products in Development

PEG-PAL is an investigational enzyme substitution therapy. It is being developed as a subcutaneous injection and is intended for those patients with PKU that do not respond to Kuvan. In preclinical models, PEG-PAL produced a dose-dependent reduction in blood phenylalanine (Phe) levels, the same endpoint that was used in the Kuvan studies. The Company is also developing GALNS, an enzyme substitution therapy for the treatment of MPS IV.