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Last €106.67 EUR
Change Today -0.71 / -0.66%
Volume 340.0
BM8 On Other Exchanges
As of 11:10 AM 04/28/15 All times are local (Market data is delayed by at least 15 minutes).

biomarin pharmaceutical inc (BM8) Snapshot

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52 Week High
03/31/15 - €123.02
52 Week Low
05/21/14 - €40.56
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Dividend Yield

biomarin pharmaceutical inc (BM8) Details

BioMarin Pharmaceutical Inc. develops and commercializes pharmaceuticals for serious diseases and medical conditions in the United States, Europe, Latin America, and internationally. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with mucopolysaccharidosis VI; Kuvan, a proprietary synthetic oral form of 6R-BH4 used to treat patients with phenylketonuria (PKU), an inherited metabolic disease; Aldurazyme used for the treatment of patients with mucopolysaccharidosis I, a genetic disease; Firdapse, a form of 3,4-diaminopyridine used for the treatment of Lambert Myasthenic Syndrome, an autoimmune disease. The company also conducts clinical trials on several investigational product candidates for the treatment of various diseases, including drisapersen, an exon-51 skipping compound for the treatment of Duchenne muscular dystrophy (DMD); pegvaliase, an enzyme substitution therapy for the treatment of PKU; reveglucosidase alfa, an enzyme replacement therapy for Pompe disease; talazoparib, an orally available poly-ADP ribose polymerase inhibitor for cancer treatment; BMN 111, a peptide therapeutic for achondroplasia treatment; BMN 044, BMN 045, and BMN 053 for DMD treatment; and cerliponase alfa for the treatment of late infantile neuronal ceroid lipofuscinosis. In addition, it develops preclinical product candidates for genetic and other metabolic diseases, such as BMN 270 and BMN 250. The company serves specialty pharmacies and end-users, such as hospitals and foreign government agencies; and distributors and pharmaceutical wholesalers. S.A. BioMarin Pharmaceutical Inc. has a collaboration agreement with Genzyme Corporation; and an agreement with Merck Serono. The company was founded in 1996 and is headquartered in San Rafael, California.

1,681 Employees
Last Reported Date: 03/2/15
Founded in 1996

biomarin pharmaceutical inc (BM8) Top Compensated Officers

Chief Executive Officer and Director
Total Annual Compensation: $916.0K
Chief Financial Officer and Executive Vice Pr...
Total Annual Compensation: $480.6K
Chief Medical Officer and Executive Vice Pres...
Total Annual Compensation: $528.2K
Chief Commercial Officer and Executive Vice P...
Total Annual Compensation: $417.7K
Senior Vice President, General Counsel and Se...
Total Annual Compensation: $409.6K
Compensation as of Fiscal Year 2014.

biomarin pharmaceutical inc (BM8) Key Developments

BioMarin Pharmaceutical Inc. Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc. announced completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen, an investigational exon-skipping drug candidate for the treatment of the large genetically defined subset of Duchenne muscular dystrophy (DMD). DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with about 20,000 new cases diagnosed globally each year. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients. The company intends to also submit an application for registration in the European Union in summer 2015. Drisapersen has been granted Orphan and Fast Track status, as well as Breakthrough Therapy designation by the FDA.

BioMarin Pharmaceutical Inc. Announces Results of Natural History Study to Evaluate the Progression of Patients with Duchenne Muscular Dystrophy at the 67th American Academy of Neurology Annual Meeting

BioMarin Pharmaceutical Inc. announced the results of a natural history study designed to prospectively evaluate the progression of patients with Duchenne muscular dystrophy (DMD) at the 67th American Academy of Neurology (AAN) Annual Meeting in Washington, D.C., April 18-25, 2015. This observational study of nearly 270 pediatric patients (between three and 18 years of age) was designed to characterize the progression of DMD over a three year period to provide information that may help in the development of therapeutic clinical trials. The results of the 12-month interim analysis of 77 ambulatory patients further describes the relationship between baseline characteristics, including age and six-minute walking distance and the trajectory of disease progression. These findings are consistent with that reported in other natural history studies. Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12 years. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure. PRODMD-01 (NCT01753804) is a prospective, multi-center, exploratory, observational study of physical impairment, activity limitation and quality of life in young males with Duchenne muscular dystrophy, resulting from a mutation of the DMD gene. No medication or treatment are being evaluated in this study. A total of 269 males, between the ages of three and 18 years and a life expectancy of greater than three years, were enrolled in the study. Investigators are monitoring the subjects as they perform various physical tests, and complete quality of life questionnaires to help with determining how this condition progresses over time. Patients are to be assessed every six months over a period of three years. Certain biomarkers are measured in blood and urine samples to investigate a possible relation with disease progression.

Biomarin Pharmaceutical Inc. Announces the Results of a Post-Hoc Sub-Analysis of the PKU-016 or ASCEND Study

BioMarin Pharmaceutical Inc. announced the results of a post-hoc sub-analysis of the PKU-016 or ASCEND study, the large randomized controlled trial evaluating neurocognitive outcomes in patients with phenylketonuria (PKU) treated with the approved therapy Kuvan® (sapropterin dihydrochloride) at the 2015 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting. The sub-analysis included 86 subjects 8 to 17 years of age with PKU who were randomized to blinded treatment with Kuvan (N = 43) or placebo (N = 43) for 13 weeks, after which all individuals received open label Kuvan for an additional 13 weeks. This analysis evaluated the effects of Kuvan treatment on blood phenylalanine (Phe) concentration, attention deficit hyperactivity disorder like symptoms, and executive function defects in children and adolescents with PKU. Symptoms of ADHD were evaluated by using the attention deficit hyperactivity rating scale (ADHD-RS) commonly used to evaluate symptoms of inattentiveness and hyperactivity. There was a statistically significant difference between the baseline and week 13 ADHD-RS total score with Kuvan compared with placebo (p = 0.01). In addition to the total ADHD-RS score, the sub-analysis showed a statistically significant (p= 0.04) improvement in the inattention subscale score and statistically significance (p= 0.016) improvement in the hyperactivity subscale score for the Kuvan group compared with placebo from baseline to week 13.


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Price/Sales 23.7x
Price/Book 11.7x
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TEV/Sales 23.5x

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