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BioMarin Pharmaceuticals Inc. (symbol: BMRN) is a global biotechnology firm dedicated to the development and commercialization of first-in-class and best-in-class therapeutics. Founded in 1997, the company's mission is to transform the lives of patients suffering from serious and life-threatening rare genetic diseases, particularly those affecting children. BioMarin targets rare conditions that are often inherited, difficult to diagnose, progressively debilitating, and lack effective treatment options.
BioMarin's diverse product portfolio includes independently marketed therapies such as Naglazyme, Vimizim, and Brineura, as well as Kuvan and Palynziq for treating phenylketonuria (PKU). The company also collaborates with Genzyme to market Aldurazyme. In 2021, BioMarin's Voxzogo (vosoritide) was approved for the treatment of achondroplasia, a rare genetic disorder affecting bone growth. Following this, in 2022, Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for hemophilia A, received approval in Europe and in the U.S. in 2023.
BioMarin places significant emphasis on research, development, manufacturing, and commercialization speed, aiming to deliver important therapies promptly. The company's capabilities have led to numerous transformational therapies addressing unmet medical needs, providing substantial benefits over existing treatments.
Notable recent developments include the expanded FDA approval for Voxzogo to increase linear growth in pediatric patients of all ages with achondroplasia with open epiphyses, and the substantial revenue growth contributed by global demand for Voxzogo. BioMarin also focuses on hypochondroplasia, launching a pivotal clinical trial program and presenting positive early results from a Phase 2 study.
Financially, BioMarin has demonstrated robust growth, with increased revenue driven by high demand for its products. The company has secured agreements with U.S. and European health authorities to expand patient access to Roctavian and other treatments. Strategic initiatives include corporate governance enhancements and collaborations with major investors to maximize long-term value creation.
For further details about ongoing clinical trials, product information, and patient support, visit www.biomarin.com.
BioMarin Pharmaceutical (NASDAQ: BMRN) reported robust financial results for Q4 and FY 2024, with total revenues of $747 million for Q4 (+16% Y/Y) and $2.85 billion for the full year (+18% Y/Y). The growth was driven by strong contributions from VOXZOGO and Enzyme Therapies. GAAP net income for Q4 increased by $105 million to $125 million, while non-GAAP income rose by $85 million to $180 million. The company projects double-digit revenue and profitability growth in 2025, with total revenues expected to grow by 10% Y/Y.
Key developments include the advancement of pipeline candidates such as BMN 351 for Duchenne Muscular Dystrophy and BMN 333 for skeletal conditions, with significant clinical data expected in 2025. VOXZOGO saw a 42% increase in Q4 and 56% in FY 2024, and is on track to be available in over 60 countries by 2027.
BioMarin's full-year GAAP operating margin expanded by 9.3 percentage points to 17.0%, with a GAAP diluted EPS of $2.21 (+154% Y/Y). The company expects a non-GAAP operating margin of 40% by 2026. Operating cash flows for FY 2024 totaled $573 million, a 260% increase Y/Y, with total cash and investments at $1.7 billion. BioMarin targets over $1.25 billion in operating cash flows annually starting in 2027.
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%.
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