Welcome to our dedicated page for Biomarin Pharmaceutical news (Ticker: BMRN), a resource for investors and traders seeking the latest updates and insights on Biomarin Pharmaceutical stock.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is a global rare disease biotechnology company focused on medicines for genetically defined conditions. This news page aggregates company announcements, press releases and event updates so readers can follow how BioMarin’s strategy, pipeline and commercial portfolio evolve over time.
Recent communications from BioMarin highlight its emphasis on Enzyme Therapies and Skeletal Conditions business units, as well as its work with ROCTAVIAN, a gene therapy for hemophilia A. The company issues news on topics such as financial results, long-term guidance, corporate strategy, business development transactions, clinical data presentations and regulatory milestones for its therapies.
Examples of news themes include updates on VOXZOGO in achondroplasia and other skeletal conditions, progress with PALYNZIQ in phenylketonuria, development of BMN 401 for ENPP1 deficiency and other pipeline programs in rare genetic diseases. BioMarin also reports on strategic partnerships, such as digital and data collaborations, and on planned acquisitions intended to expand and diversify its rare disease portfolio.
Investors and followers of BMRN can use this page to review earnings announcements, conference presentations, investor day materials and product-specific updates released through newswires. By checking this feed regularly, readers can see how BioMarin describes its growth priorities, clinical milestones and capital allocation decisions across its rare disease franchises.
BioMarin Pharmaceutical (NASDAQ: BMRN) has scheduled its fourth quarter and full-year 2024 financial results conference call and webcast for Wednesday, February 19, 2025, at 4:30 p.m. ET. The event will be hosted by Alexander Hardy, President and Chief Executive Officer, who will discuss financial results and provide a general business update.
Participants can join via phone using U.S./Canada dial-in (800-715-9871) or International dial-in (646-307-1963) with Conference ID 1878833. A live audio webcast will be available through BioMarin's investor website. A replay will be accessible for one week following the call, with U.S./Canada replay number (800-770-2030) and International replay number (609-800-9909), Playback ID 1878833.
BioMarin Pharmaceutical (Nasdaq: BMRN) has initiated legal action against Ascendis Pharma A/S in the Unified Patent Court (UPC) in Munich, Germany, for alleged infringement of European patent EP 3 175 863 B1. The patent, which covers long-acting variants of C-Type Natriuretic Peptide (CNP), was confirmed by the European Patent Office's Opposition Division in 2024.
The legal action targets Ascendis' TransCon CNP investigational product and its development program in Germany and Europe. A decision is expected within 12-15 months. The UPC, operational since June 2023, has jurisdiction over European patents across 18 European member states that have ratified the Unitary Patents.
BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its participation in the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, California. Alexander Hardy, the company's President and Chief Executive Officer, will deliver a presentation on Monday, January 13, 2024, at 9:00 am PT / 12:00 pm ET.
The presentation will be accessible through a live audio webcast on the company's investor relations website. For those unable to attend or watch live, an archived version of the presentation will be available for a time through BioMarin's website.
BioMarin Pharmaceutical (Nasdaq: BMRN) presented positive real-world evidence for VOXZOGO in treating children with achondroplasia at the ESPE Meeting 2024. Data from the European CrescNet registry, involving 452 children across 30 centers, showed significant height improvements. After 12 months of treatment, 143 participants averaged a 6.36 cm height increase, while 73 participants treated for 24 months showed an 11.86 cm increase. A separate French study of 17 children above age 5 demonstrated an average 8.76 cm height increase over 18 months, with no treatment discontinuations. The company has collected over 6,000 patient-years of safety data through their CANOPY clinical program.
BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its participation in three major investor conferences in November and December 2024. The company will present at the UBS Global Healthcare Conference on November 12 at 11:00 am PST, the Jefferies London Healthcare Conference on November 19 at 9:00 am GMT, and the 7th Annual Evercore ISI HealthCONx Conference on December 4 at 7:00 am PST.
Live audio webcasts of the presentations will be accessible through BioMarin's investor website, with archived versions available for -time replay.
BioMarin Pharmaceutical reported strong Q3 2024 financial results with total revenues of $746 million, representing a 28% year-over-year growth. VOXZOGO showed exceptional performance with 54% revenue growth, while Enzyme Therapies portfolio increased by 27%. The company's GAAP Diluted EPS reached $0.55 (+162% Y/Y), and Non-GAAP Diluted EPS was $0.91 (+98% Y/Y).
Over 3,800 children globally are now receiving VOXZOGO treatment for achondroplasia, with majority of new U.S. patient starts being under 5 years of age. The company increased its full-year 2024 guidance and reaffirmed its long-term outlook, targeting $4 billion in Total Revenues by 2027.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that its President and CEO, Alexander Hardy, will host a conference call and webcast on Tuesday, October 29, 2024, at 4:30 p.m. ET to discuss the company's third quarter 2024 financial results and provide a general business update. The event will be accessible via phone and webcast, with dial-in numbers provided for both U.S./Canada and international participants. A replay of the call will be available, and interested parties can access a live audio webcast through the investor section of BioMarin's website. The replay will be archived on the site for one week following the call.
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) will present positive data from the CANOPY clinical program evaluating VOXZOGO® (vosoritide) at the 16th International Skeletal Dysplasia Society meeting. The data shows:
- Meaningful improvements in health-related quality of life for children with achondroplasia
- Increased bone length while maintaining bone strength
- Sustained growth gains in children with hypochondroplasia, Noonan syndrome, and genetic variants associated with idiopathic short stature
VOXZOGO is becoming the standard of care in achondroplasia, with proven effects on growth velocity, safety profile, and positive impact on proportionality and quality of life. The drug shows promise in treating other genetic skeletal conditions, with ongoing research demonstrating efficacy and safety consistent with its profile in achondroplasia.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) hosted an Investor Day, unveiling a new corporate strategy and financial guidance for 2027. The company aims to deliver sustained value creation through a restructured organization built around three business units: Enzyme Therapies, Skeletal Conditions, and ROCTAVIAN®. BioMarin plans to implement a $500 million cost transformation program to support Non-GAAP Operating margin targets.
Key financial projections include:
- Total Revenues of $4 billion by 2027
- Non-GAAP Operating Margin in the low-to-mid 40%s starting with 40% in 2026
- Operating Cash flow of $1.25 billion+ by 2027
The company reaffirmed its 2024 guidance, projecting Total Revenues of $2.750B to $2.825B and Non-GAAP Operating Margin of 26% to 27%.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is hosting its Investor Day on September 4, 2024, in New York, starting at 10:30 a.m. Eastern Time. The event, led by President and CEO Alexander Hardy, will showcase the company's new strategic priorities aimed at delivering significant value creation.
Key presentations include:
- BioMarin's new corporate strategy for sustained value creation
- Financial strategy for high growth and superior returns
- Innovation strategy for a sustainable pipeline of high-impact medicines
- Commercial growth strategy, focusing on enzyme therapies and leadership in achondroplasia
The event will be accessible virtually, with a live webcast, replay, and presentation slides available on BioMarin's Investor Relations website for at least 12 months.