BioMarin Pharmaceutical

BioMarin Pharmaceutical, Inc. engages in the development and commercialization of therapies for people with serious and life-threatening rare diseases and medical conditions. Its pipeline products include Valoctocogene roxaparvovec, Vosoritide, and BMN 307. The company was founded by John C. Klock, Christopher M. Starr and Grant W. Denison on March 21, 1997, and is headquartered in San Rafael, CA.

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BioMarin excels in Q4 with record revenues and gene therapy launch

BioMarin Pharmaceutical reported strong financial results in Q4 2022, achieving revenue growth, profitability, and successful introduction of the ROCTAVIAN gene therapy for hemophilia A.
The company's performance highlights its R&D capabilities, market positioning, and strategic readiness for upcoming product launches, positioning BioMarin for continued growth and innovation.
While facing challenges in reimbursement processes and regulatory requirements, BioMarin has opportunities to drive revenue expansion, market penetration, and address unmet medical needs through its innovative pipeline.

BioMarin reports strong Q1 earnings, eyes blockbuster status for ROCTAVIAN

BioMarin Pharmaceuticals had a robust Q1 2023, focusing on revenue growth, profitability, and commercial readiness for key drug launches. While facing challenges in Germany, the company is optimistic about the potential of ROCTAVIAN and VOXZOGO in driving future growth.

BioMarin excels in Q2 with record revenues and treatment milestones

BioMarin's strong Q2 performance, driven by record revenues and product demand, positions the company for growth.
Challenges like supply issues and site readiness are being addressed, while opportunities in new programs and markets are promising.
Overall, BioMarin's focus on compliance, expansion, and pipeline development sets a positive trajectory for future success.

BioMarin reports strong Q3 revenue growth, eyes Roctavian expansion

BioMarin's Q3 performance showed revenue growth driven by Voxzogo, with strong expectations for 2024 revenue.
New CEO Alexander Hardy is set to continue the core strategy, focusing on Roctavian's global launch and Voxzogo's growth potential.
Challenges include delays in Roctavian's takeoff and supply constraints for Voxzogo, but efforts are underway to address these issues.
Opportunities lie in Roctavian's revenue contribution, Voxzogo's U.S. growth potential, and leveraging CNP for stature improvements.

BioMarin posts robust Q4 earnings, eyes expansion in gene therapy

BioMarin Pharmaceutical delivered strong financial results in Q4 2023, driven by revenue growth and increased earnings per share. The company's focus on high-impact medicines and gene therapy expertise positions it well for future growth.
While facing challenges related to analyst inquiries and market adoption of key products like Roctavian and Voxzogo, BioMarin remains committed to innovation and optimizing its business model.
Opportunities lie in expanding product reach, maximizing shareholder value through strategic capital allocation, and driving innovation in transformative programs. BioMarin's long-term strategy and growth potential remain promising.

BioMarin reports record revenue, focuses on key assets for growth

BioMarin Pharmaceutical's Q1 2024 performance reflects strong revenue growth and financial stability, driven by key product demand.
While facing challenges such as revenue impact and supply constraints, the company's strategic focus on key assets and cost reduction initiatives bode well for future growth.
Opportunities lie in expanding Voxzogo's market reach, optimizing manufacturing capabilities, and maintaining a competitive edge in innovative treatments.

BioMarin Pharmaceuticals reports record revenue and profitability outlook for 2024

BioMarin Pharmaceuticals delivered strong financial performance in Q2 2024, driven by robust revenue growth and strategic decisions. The company's focus on profitability, expansion of key treatments, and progress in R&D highlight a promising outlook. Addressing analyst inquiries and leveraging upcoming events can further enhance investor confidence and market positioning.

BioMarin reports record Q3 revenues, eyes long-term growth targets

BioMarin Pharmaceuticals showcased robust financial performance and raised full-year guidance.
The company remains optimistic about its long-term growth prospects and is committed to defending its IP.
Strategic focus on expanding global reach and driving growth in enzyme therapies portfolio.

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The mean of analysts' price targets for BioMarin (BMRN) points to a 30.9% upside in the stock. While this highly sought-after metric has not proven reasonably effective, strong agreement among analysts in raising earnings estimates does indicate an upside in the stock.

Wall Street Analysts See a 31% Upside in BioMarin (BMRN): Can the Stock Really Move This High?

BioMarin (BMRN) intends to reduce the existing workforce by nearly 4%. The company plans to use the savings in costs and redirect them toward its operating expenses.

BioMarin (BMRN) Starts Restructuring Moves to Increase Efficiency

SAN RAFAEL, Calif. , Oct. 6, 2022 /PRNewswire/ -- 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 success, prepare for a potential launch of ROCTAVIAN™ (valoctocogene roxaparvovec) in the U.S., and drive core infrastructure optimization.

BioMarin Simplifies Organizational Structure to Increase Efficiency

SAN RAFAEL, Calif. , Oct. 4, 2022 /PRNewswire/ -- 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, October 26 th, at 4:30 p.m.

BioMarin to Host Third Quarter 2022 Financial Results Conference Call and Webcast on Wednesday, October 26, at 4:30pm ET

BioMarin Pharmaceutical's revenue was $533.8 million in Q2 2022, up 6.4% year-on-year. The company's net income has been growing at a substantial pace in recent quarters, helping to keep BioMarin Pharmaceutical's business profitable.

BioMarin Pharmaceutical: Leader In Rare Disease Innovation

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If approved, BioMarin's (BMRN) valoctocogene roxaparvovec will be the first gene therapy for hemophilia A in the United States. The FDA filing also incorporates BMRN's responses to the CRL issued to the BLA in 2020.

BioMarin (BMRN) Refiles BLA With FDA for Hemophilia Gene Therapy

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. , Sept. 29, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Company resubmitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.

BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA

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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) Resubmission to FDA on Track for End of September 2022 SAN RAFAEL, Calif. , Sept. 15, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Institute for Clinical and Economic Review (ICER) released a Draft Evidence Report updating the previous Hemophilia A assessment of the comparative clinical effectiveness and value of ROCTAVIAN ™ (valoctocogene roxaparvovec).

BioMarin's Gene Therapy for Adults with Severe Hemophilia A, ROCTAVIAN™ (valoctocogene roxaparvovec), Assessed to Provide Substantial Cost Savings Per Patient in a Preliminary Independent Report

BioMarin (BMRN) reported earnings 30 days ago. What's next for the stock?

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BioMarin's (BMRN) Roctavian is the first gene therapy to get approval for hemophilia A in the European Union. The therapy has also been granted a 10-year market exclusivity.

BioMarin's (BMRN) Hemophilia Gene Therapy Gets Nod in EU

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 Determination of ODD Conference Call and Webcast to be Held Wed., Aug. 24th at 8:00 pm Eastern SAN RAFAEL, Calif.

First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec), Approved by European Commission (EC)

NEW ORLEANS , Aug. 12, 2022 /PRNewswire/ -- Former Attorney General of Louisiana, Charles C. Foti, Jr., Esq.

BIOMARIN INVESTIGATION CONTINUED BY FORMER LOUISIANA ATTORNEY GENERAL: Kahn Swick & Foti, LLC Continues to Investigate the Officers and Directors of BioMarin Pharmaceutical Inc. - BMRN

Biomarin Pharmaceutical's (BMRN) second-quarter earnings and sales beat estimates. BMRN slightly raises its sales guidance for the year.

BioMarin's (BMRN) Q2 Earnings Top, Voxzogo Drives Sales

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN ) Q2 2022 Earnings Conference Call August 3, 2022 4:30 PM ET Company Participants Traci McCarty - Vice President of Investor Relations J.J. Bienaime - Chairman and Chief Executive Officer Jeff Ajer - Executive Vice President and Chief Commercial Officer Hank Fuchs - President, Worldwide Research and Development Brian Mueller - Executive Vice President and Chief Financial Officer Conference Call Participants Operator Welcome to the BioMarin Second Quarter 2022 Financial Results Conference Call.

BioMarin Pharmaceutical Inc. (BMRN) CEO J.J. Bienaime on Q2 2022 Results - Earnings Call Transcript

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 roxaparvovec) for the Treatment of Severe Hemophilia A Will Be Approved in Europe in the Third Quarter; Re-Submission of Biologics License Application (BLA) in the U.S. Planned for September 2022 Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended June 30, Six Months Ended June 30, 2022 2021 % Change 2022 2021 % Change Total Revenues $       533.8 $       501.7 6 % $    1,053.2 $       987.7 7 % Net Product Revenues Marketed by BioMarin (1) $       480.4 $       458.6 5 % $       961.5 $       876.3 10 % Vimizim Net Product Revenues $       173.3 $       171.7 1 % $       356.4 $       329.9 8 % Naglazyme Net Product Revenues $       115.8 $       118.8 (3) % $       243.8 $       226.1 8 % Palynziq Net Product Revenues $         61.6 $         59.0 4 % $       116.5 $       113.0 3 % Kuvan Net Product Revenues $         57.6 $         78.8 (27) % $       116.9 $       149.6 (22) % Brineura Net Product Revenues $         37.7 $         30.3 24 % $         73.9 $         57.7 28 % Voxzogo Net Product Revenues $         34.4 $             — n/a $         54.0 $             — n/a Aldurazyme Net Product Revenues $         37.3 $         28.1 33 % $         61.7 $         78.1 (21) % GAAP Net Income $         27.7 $         12.9 $       148.5 $         30.3 GAAP Net Income per Share – Basic $         0.15 $         0.07 $         0.80 $         0.17 GAAP Net Income per Share – Diluted $         0.15 $         0.07 $         0.79 $         0.16 Non-GAAP Income (2) $       109.4 $         97.8 $       214.6 $       202.1   June 30, 2022 December 31, 2021 Total cash, cash equivalents & investments $               1,522.3 $               1,521.7   (1) Net Product Revenues Marketed by BioMarin is the sum of revenues from Vimizim, Naglazyme, Palynziq, Kuvan, Brineura and Voxzogo for the three and six months ended June 30, 2022 and 2021, each calculated in accordance with Generally Accepted Accounting Principles in the United States (U.S. GAAP). Sanofi is BioMarin's sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third parties.

BioMarin Announces Record Revenues in Second Quarter 2022; Increases Full-year 2022 Top and Bottom-line Guidance

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BioMarin (BMRN) doesn't possess the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.

Analysts Estimate BioMarin Pharmaceutical (BMRN) to Report a Decline in Earnings: What to Look Out for

The recent Japan approval of Voxzogo for achondroplasia would generate a leaping sales growth in the coming quarters. BioMarin is also expanding Voxzogo's label for younger kids afflicted by achondroplasia.

BioMarin: More Growth

SAN RAFAEL, Calif. , July 20, 2022 /PRNewswire/ -- 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, August 3 rd, at 4:30 p.m.

BioMarin to Host Second Quarter 2022 Financial Results Conference Call and Webcast on Wednesday, August 3, at 4:30pm ET

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 Approval for Valoctocogene Roxaparvovec Expected Q3 2022 SAN RAFAEL, Calif. , July 11, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that it presented four oral presentations and two poster presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 World Congress from July 9-12 in London.

BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress July 9-12, Including 4 Oral and 2 Poster Presentations

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On Friday, BioMarin Pharmaceutical received a positive CHMP opinion in Europe for ValRox for Hemophilia type A treatment. According to the last report, the core portfolio looks solid.

BioMarin's Solid Pipeline Bears The First Fruit

BioMarin (BMRN) receives a positive CHMP opinion for Roctavian, its gene therapy for treating adults with hemophilia A. The European Commission's decision is expected by the third quarter of 2022.

News for BioMarin Pharmaceutical Stock (BMRN)