BioMarin Pharmaceutical Inc.

Our Purpose

At BioMarin, we translate the promise of genetic discovery into medicines that make a profound impact on the life of each patient.

Since our founding in 1997, we’ve developed a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we seek to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world.

As we have looked to expand our impact, our approach to cutting-edge science has remained the same. We enlist the best of the best – people with the right technical expertise and a relentless drive to solve real problems – and create an environment that empowers our teams to pursue bold, innovative science.

770 Lindaro Street
San Rafael, CA 94901

105 Digital Drive
Novato, CA 94949

Tel: (415) 506-6700

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NEWS

BioMarin's headquarters in San Francisco

FDA

BioMarin Wins FDA Approval for First Gene Therapy for Hemophilia A

After an initial rejection, BioMarin has finally secured the FDA’s approval for Roctavian, the first gene therapy in the U.S. for the most common form of the bleeding disorder.

June 30, 2023

2 min read

Tristan Manalac

FDA Headquarters_Grandbrothers/Adobe Stock

Drug Development

FDA Action Alert: Regeneron, BioMarin, Eton and Amneal

Regeneron envisions new dosing for Eylea’s label and it’s the moment of truth for BioMarin’s gene therapy for hemophilia A. For that and more, see inside.

June 23, 2023

4 min read

Tristan Manalac

Drug Development

Small Study of BridgeBio’s Dwarfism Drug Indicates Advantage over BioMarin

BridgeBio posted data Monday from its small but highly anticipated Phase II study of infigratinib in achondroplasia - a drug that could give BioMarin a run for its money.

March 7, 2023

2 min read

Kate Goodwin

Drug Development

FDA Adcomm Cancels Meeting on BioMarin’s Hemophilia A Gene Therapy

BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its BLA for Roctavian an AAV gene therapy for adults with severe hemophilia A.

November 23, 2022

2 min read

Rosemary Scott

Drug Development

BioMarin Faces Potential 3-Month Delay for Hemophilia A Gene Therapy (Updated)

A request for additional data may cause a 3-month delay for potential approval of BioMarin Pharmaceutical’s hemophilia A gene therapy.

November 8, 2022

2 min read

Alex Keown

Business

FDA Adcomm to Meet on BioMarin’s Hemophilia A Gene Therapy

During its third-quarter earnings report, BioMarin noted that the FDA planned to hold an advisory committee meeting to discuss the company’s gene therapy for hemophilia A.

October 28, 2022

2 min read

Mark Terry

Business

BioMarin, Cyclerion Slash Workforce in the Name of Strategic Focus

BioMarin Pharmaceuticals announced Friday it was shedding around 4% of its global workforce, or around 120 jobs, in an effort to improve its operational efficiency.

October 7, 2022

3 min read

Tristan Manalac

Drug Development

BioMarin Banks on More Robust Data in Hemophilia A Resubmission

Two years after receiving a Complete Response Letter for its hemophilia gene therapy, BioMarin is heading back to the FDA with stronger and more robust data for potential approval.

September 30, 2022

3 min read

Alex Keown

Genetown

ICER Deems Hemophilia Gene Therapies Worth the $2.5M Price Tag (Updated)

The Institute for Clinical and Economic Review (ICER) concluded that even at a hefty lifetime cost of $2.5 million, gene therapies for hemophilia A and B are worth it.

September 15, 2022

3 min read

Tristan Manalac

JOBS