Press Releases

FDA No Longer Plans to Hold an Advisory Committee Meeting, as Previously Planned, to Discuss the Biologics License Application (BLA) BioMarin Remains on Track to Host Scheduled Manufacturing...

FDA Pre-Licensure Inspection of Gene Therapy Manufacturing Facility Scheduled FDA Requests Submission of Upcoming 3-Year Data Analysis from Phase 3 GENEr8-1 Study Current PDUFA Target Action Date...

Credit Suisse 31st Annual Healthcare Conference 2022: November 8th, 8:35amPT/11:35amET, in Rancho Palos Verdes, CA 13th Annual Jefferies Global Healthcare Conference 2022: November 16th, 8:35amGMT, in

VOXZOGO® $48 Million Contribution in the Quarter Results in BioMarin Raising Full-year 2022 VOXZOGO Net Product Revenue Guidance to Between $140 Million and $170 Million U.S. Biologics...

If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A PDUFA Target Action Date is March 31, 2023 SAN RAFAEL, Calif., Oct. 12, 2022 /PRNewswire/ -- BioMarin...

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced its decision to redesign the organization to better focus investments that advance its R&D pipeline, maximize recent commercial launch...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

BLA Includes Substantial Body of Data from Pivotal Phase 3 and Ongoing Phase 1/2 Studies If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A SAN RAFAEL, Calif.,...

Institute for Clinical and Economic Review (ICER) Base-Case Model Report Results in $4 Million Savings Versus Emicizumab Prophylaxis Per Patient Over a Lifetime Biologics License Application (BLA)...

Maintains Orphan Drug Designation (ODD) in the EU Providing 10-years of Market Exclusivity Significant Benefit Over Existing Therapies for Patients with Severe Hemophilia A in EU Based on EMA...

VOXZOGO™ $34 Million Contribution Drives Total Revenues of $534 Million in the Quarter; Total Revenues Grew 13% Year-Over-Year, Excluding Kuvan BioMarin Expects that ROCTAVIAN™ (valoctocogene...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

Commitment to Advancing Care for People with Hemophilia A Demonstrated with Largest and Longest, Ongoing Clinical Development Program for any Gene Therapy in Hemophilia A European Commission...

European Commission Approval Expected Q3 2022 1st Gene Therapy for Treatment of Hemophilia A Recommended for Approval in Europe More than 20,000 Adults with Severe Hemophilia A in BioMarin...

No Lower Age Restriction for Treatment Japan Accounts for Approximately Half of the 1,500 Patient Opportunity in APAC Region SAN RAFAEL, Calif., June 21, 2022 /PRNewswire/ -- BioMarin...

Improvement in Height Z-Score and Annualized Growth Velocity Observed, Consistent with Children Over 5 Interactions with Health Authorities Planned for H2 2022 to Discuss Expanding Access to...

95% Reduction in Mean Annualized Bleed Rate (ABR) and 96% Reduction in Mean Annualized Factor VIII Usage from Baseline Through Year 6 in 6e13 vg/kg Dose Cohort 91% Reduction in Mean ABR and 93%...

Humaira Serajuddin Named to Newly Created Role of Senior Vice President, Chief Marketing Officer Erin Burkhart, Named Group Vice President, Chief Accounting Officer SAN RAFAEL, Calif., May 5, 2022...

Ongoing Clinical Development Program Represents Largest and Longest Development Program for any Gene Therapy in Hemophilia A, Demonstrates Commitment to Advancing Care for People with Hemophilia A...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at the BofA Securities Healthcare Conference on May 12...

BioMarin Reports Record First Quarter Total Revenues of $519 Million Driven by a $20 Million VOXZOGO Contribution; Total Revenues Grew 11% Year-over-Year, Excluding Kuvan Full-year 2022 Financial...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, will host a conference call and webcast on Wednesday,...

New England Journal of Medicine Publishes Editorial: Prepare the Way for Hemophilia A Gene Therapy Significantly Reduced Bleeding and Factor VIII Utilization Compared to Baseline Factor VIII...

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended December 31, Twelve Months Ended December 31, 2021 2020 % Change 2021 2020 % Change Total...

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced updates on its investigational gene therapy programs in clinical development. In February 2022, the Company received additional requests from...