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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 reported a strong financial performance for Q2 2024, with total revenues reaching $712 million, a 20% increase year-over-year (Y/Y). The company also raised its full-year 2024 guidance. Key drivers include strong global demand for VOXZOGO, which saw a 73% Y/Y increase in the number of children receiving treatment, and robust performance of enzyme therapies. GAAP diluted EPS for the quarter was $0.55, a 90% Y/Y increase, while Non-GAAP diluted EPS was $0.96, up 78% Y/Y.
VOXZOGO contributions were significant, with nearly 900 new patient starts in the first half of 2024. BioMarin's enzyme therapies also showed strong growth, partially offset by lower revenues from KUVAN due to generic competition. GAAP net income for Q2 2024 increased by $51.2 million to $107.2 million. Non-GAAP income rose by $83.7 million to $188.9 million. Financial guidance for 2024 was updated to reflect these strong results.
BioMarin continued to make progress in its clinical programs and strategic priorities, including its supply chain efforts and development of new indications for VOXZOGO.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced a strategic shift for its hemophilia A treatment, ROCTAVIAN®. The company will focus commercial operations on the United States, Germany, and Italy, where the medicine is approved and reimbursed. This move aims to reduce annual direct ROCTAVIAN expenses to approximately $60 million by 2025, with the goal of achieving profitability for the product by the end of that year.
Key changes include:
- Reducing investments in development and manufacturing
- Focusing on markets where ROCTAVIAN is approved and reimbursed
- Placing the gene therapy manufacturing facility in an idle state
- Discontinuing enrollment in new clinical development programs
- Continuing support for patients already treated with ROCTAVIAN
BioMarin remains committed to ROCTAVIAN as an important option for people with severe hemophilia A, citing its potential for years of bleed control after a single treatment.
The FDA has approved BioMarin's supplemental Biologics License Application (sBLA) for BRINEURA® (cerliponase alfa) to treat children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), including those under 3 years, regardless of symptom presence. Previously, BRINEURA was only indicated for symptomatic children aged 3 and above. The approval is based on data from Study 190-203, a Phase 2 trial that showed BRINEURA slows motor function decline and delays disease onset in children aged 1-6, including those under 3. In this study, none of the treated children under 3 experienced a significant motor decline, compared to 61% of untreated children in a natural history cohort. The safety profile for children under 3 is consistent with the known safety profile of the drug. This expanded indication emphasizes the importance of early diagnosis and treatment for CLN2 disease.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that it will host a conference call and webcast on Monday, August 5, 2024, at 4:30 p.m. ET to discuss its second quarter 2024 financial results and provide a general business update. The call will be hosted by Alexander Hardy, President and Chief Executive Officer of BioMarin.
Participants can join the call using the following details:
- U.S. / Canada Dial-in: 888-596-4144
- International Dial-in: 646-968-2525
- Conference Call ID: 1816377
BioMarin announced new data from its Phase 2 111-205 study showing that children with achondroplasia treated with VOXZOGO® exhibited significant increases in bone length while maintaining bone strength over five years. The results will be presented at the International Conference on Children's Bone Health in June 2024. Additional Phase 3 data reveal VOXZOGO's positive impacts on proportionality and health-related quality of life. Separately, research on hypochondroplasia highlights higher comorbidity rates, underscoring unmet medical needs. BioMarin is advancing clinical trials for hypochondroplasia, idiopathic short stature, and other growth conditions.
BioMarin presented four-year data from its Phase 3 study on ROCTAVIAN® at the ISTH 2024 Congress, showing long-term safety and efficacy in treating severe hemophilia A. The study revealed that 73.6% of patients had zero treated bleeds in year four, with stable FVIII levels. Mean FVIII activity was 27.1 and 16.1 IU/dL by OSA and CSA assays, respectively. Additionally, there was a notable improvement in health-related quality of life (HRQoL), with a 6.2 point increase in Haemo-QOL-A Total Score. Phase 2 results also showed seven-year bleed control for a majority of patients.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will be presenting at the BofA Securities 2024 Healthcare Conference on May 15, 2024, in Las Vegas, NV. Alexander Hardy, President, and CEO of BioMarin, will be presenting at 10:00am PT / 1:00pm ET. The presentation will be available via live audio webcast and archived on the company's website.
BioMarin Pharmaceutical Inc. presented positive new data at the 2024 Pediatric Endocrine Society Annual Meeting, highlighting the safety and efficacy of VOXZOGO® (vosoritide) in children with achondroplasia and other growth-related conditions. Results from multiple studies showed significant improvements in annualized growth velocity and height standard deviation, particularly in children with Noonan syndrome, ACAN deficiency, and NPR2 mutations. Data also demonstrated VOXZOGO's potential to positively impact growth beyond achondroplasia. Clinical trials for growth-related conditions are ongoing, with promising results for children with achondroplasia who initiated treatment during adolescence.
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