Noonan Syndrome

Third Quarter 2024 Total Revenues of $746 million (+28% Y/Y and +32% at Constant Currency Y/Y); Year-to-date 2024 Total Revenues of $2.11 billion (+19% Y/Y and +23% at Constant Currency Y/Y) During the Quarter, Strong Demand Drove 54% Y/Y Revenue Growth for VOXZOGO® During the Quarter, Revenues from Enzyme Therapies Portfolio Increased 27% Y/Y Third Quarter 2024 GAAP Diluted Earnings Per Share (EPS) of $0.55 (+162% Y/Y); Year-to-date 2024 GAAP Diluted EPS of $1.56 (+103% Y/Y) Third Quarter 2024 Non-GAAP Diluted EPS of $0.91 (+98% Y/Y); Year-to-date 2024 Non-GAAP Diluted EPS of $2.60 (+63% Y/Y) Conference Call and Webcast Scheduled Today at 4:30 p.m. ET SAN RAFAEL, Calif., Oct. 29, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the quarter and nine months ended September 30, 2024. "The strategic and operational decisions we have made over the last nine months are driving strong performance, reflected in year-over-year revenue growth in the third quarter of 28% and accelerated profitability," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We are executing on our new corporate strategy, focused on Innovation, Growth, and Value Commitment, as demonstrated by another quarter of strong financial performance and clinical progress as we advance potential new medicines for the patients we serve." Mr. Hardy added, "Strong global demand for VOXZOGO led to over 3,800 infants and children receiving treatment as of the end of the third quarter. In the U.S., the majority of new patient starts in the third quarter were for children under 5 years of age, reflecting strong adoption from families seeking VOXZOGO therapy for their infants and young children. Our Enzyme Therapies portfolio also delivered impressive results, with third quarter revenue growth of 27% year-over-year, evidence of the continued growth potential of this portfolio," added Mr. Hardy. "Additionally, we made good progress advancing all of our innovative therapies under development. Within our CANOPY clinical program, we advanced our studies in five new indications with VOXZOGO. For hypochondroplasia, we are on track to complete enrollment in the Phase 3 registration-enabling study in the first half of 2025." Financial Highlights: Total Revenues for the third quarter of 2024 were $746 million, an increase of 28%, compared to the same period in 2023, driven by strong VOXZOGO contributions from new patient starts in all regions. In the quarter, revenues from BioMarin's Enzyme Therapies (VIMIZIM®, NAGLAZYME®, ALDURAZYME®, BRINEURA® and PALYNZIQ®) increased 27% compared to the third quarter of 2023. The increase was driven by a combination of the timing of order fulfillment to Sanofi as the company recognizes ALDURAZYME revenues when the product is released and control is transferred to Sanofi, increased patient demand, and the timing of large government orders in certain regions outside the U.S. Partially offsetting the increase were lower KUVAN® product revenues attributed to continued generic competition as a result of the loss of market exclusivity in 2022. GAAP Net Income increased by $66 million to $106 million in the third quarter of 2024 compared to the same period in 2023. The increase was primarily due to higher gross profit driven by the factors noted above. The increase was partially offset by higher spend in Selling, General and Administrative (SG&A), primarily due to severance and other restructuring costs associated with organizational redesign efforts executed during the third quarter of 2024, higher income tax expense and the impact of ROCTAVIAN® inventory reserves on Cost of Sales. Non-GAAP Income increased by $89 million to $178 million in the third quarter of 2024 compared to the same period in 2023. The increase in Non-GAAP Income was primarily due to higher gross profit and lower SG&A expenses primarily related to sales and marketing activities for ROCTAVIAN outside of the U.S., Germany and Italy as the company executes on its updated strategy to focus commercial launch efforts on those three countries. The increase was partially offset by higher income tax expense and the impact of ROCTAVIAN inventory reserves on Cost of Sales. 3Q'24 Execution on New Corporate Strategy: Innovation, Growth, and Value Commitment Innovation Skeletal Conditions: During the quarter, BioMarin advanced development across its CANOPY clinical program with VOXZOGO (vosoritide) in idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency, with the pivotal study in hypochondroplasia expected to complete enrollment in the first half of 2025. BioMarin's long-acting C-type natriuretic peptide (CNP), BMN 333, remains on track for initiation of the first-in-human study in early 2025. At the 16th International Skeletal Dysplasia Society meeting (ISDS) in September, BioMarin and its external research partners contributed to eight presentations (including four orals) discussing the value of vosoritide and the burden of illness in achondroplasia and related disorders. These data included showing that children with achondroplasia treated with VOXZOGO experienced meaningful improvements in addition to height, such as gains in health-related quality of life (HRQoL), and increased bone length while maintaining bone strength. Researchers also presented encouraging data from ongoing investigator-led studies of treatment in children with other genetic skeletal conditions, including hypochondroplasia and Noonan syndrome, as well as those with genetic variants often associated with idiopathic short stature such as aggrecan (ACAN) deficiency and heterozygous NPR2 mutations. Other Clinical Pipeline Programs: With BMN 351, BioMarin's next generation oligonucleotide for Duchenne Muscular Dystrophy, the program has completed enrollment into the first dose cohort and initial proof-of-concept data is expected in 2025 (including muscle dystrophin levels after 25 weeks of dosing). With BMN 349, an oral therapeutic for Alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the program completed the single-ascending dose (SAD) phase of the first-in-human study and is expected to start dosing the multiple-ascending dose (MAD) phase of the study by end of the year. Enrollment is complete in the phase 3 study with PALYNZIQ in adolescents ages 12-17, and the study is on track for data readout in 2025 to support a potential U.S. Supplemental Biologics License Application (sBLA) in the second half of the year. Pre-clinical Programs: With BMN 390, a compound for phenylketonuria which may lower hypersensitivity and enhance exposure, an IND is expected to be submitted in the second half of 2025. With BMN 370, a targeted nanobody for the prevention of bleeding in patients with low levels of von Willebrand factor levels, the company is progressing with pre-clinical work and targeting a potential IND submission for the second half of 2025. Growth As of the end of the third quarter, over 3,800 children globally, many from infancy, were receiving VOXZOGO for the treatment of achondroplasia. VOXZOGO's broad label has been especially important to those families pursuing maximum therapeutic benefit by beginning therapy at an early age. In the U.S., the largest single market opportunity, the majority of new patient starts in the quarter were for children under the age of 5 years. VOXZOGO's extensive safety and efficacy profile led more families to begin therapeutic intervention early to potentially impact craniofacial volume, foramen magnum area, body proportionality and quality of life, in addition to durable increases in growth velocity. Achondroplasia represents a global 24,000 total addressable patient population (TAPP). While the U.S. is the largest single market opportunity, markets outside of the U.S. represent approximately 90% of eligible patients. BioMarin is in the process of pursuing VOXZOGO access into more than 20 additional countries by 2027, providing the opportunity for even more children of all ages to benefit from the only approved medicine for the treatment of achondroplasia. Enzyme Therapies continue to be a significant driver of growth, with revenues increasing 13% year-to-date, compared to the same period in 2023. As outlined at Investor Day, BioMarin is implementing new initiatives to drive sustained growth of the Enzyme Therapies across the approximately 80 countries where these medicines are available. Value Commitment During the quarter, the company made significant progress executing its financial strategy to deliver on its value commitment to stakeholders. Year-to-date, BioMarin's GAAP Operating Margin of 15.3% expanded 6.4 percentage points Y/Y and Non-GAAP Operating Margin of 27.7% expanded 7.6 percentage points Y/Y while GAAP Diluted EPS of $1.56 increased 103% Y/Y and Non-GAAP Diluted EPS of $2.60 increased 63% Y/Y. These measures of profitability increased at rates faster than revenue growth, representing the company's focus on operational efficiency. During the quarter, the company continued to benefit from its ongoing $500 million cost transformation program announced at Investor Day through the impact of prioritized program decisions and ongoing execution of the enterprise-wide reorganization. The company generated operating cash flows totaling $221 million in the third quarter, an increase of 63% compared to the same period last year. Total cash and investments at the end of the third quarter were approximately $1.5 billion, and with its increasing profitability, BioMarin is positioned to generate increasing operating cash flow into the future. In addition, BioMarin settled $495 million of convertible debt in cash during the quarter as planned, resulting in the retirement of approximately four million potentially dilutive shares. This was the first time that BioMarin retired a convertible note without issuing a new convertible instrument, thereby returning value to shareholders. Today, BioMarin increased full-year 2024 guidance for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS. The updated guidance highlights the sustained strong demand for VOXZOGO and growth trajectory of Enzyme Therapies, as well as BioMarin's commitment to expand profitability while investing in innovation. During the quarter, BioMarin reaffirmed long-term guidance and outlook previously provided at Investor Day on September 4, 2024, targeting: Approximately $4 billion in Total Revenues in 2027; 40% Non-GAAP Operating Margin(1) starting in 2026 and growing to the low- to mid-40% range over time; More than $1.25 billion operating cash flow per year starting in 2027; Mid-teen compound annual growth rate (CAGR) for total revenues through 2034; and Treatments for Skeletal Conditions to represent a greater than $5 billion revenue opportunity over time. Forward-Looking Non-GAAP Financial Information BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors. 2024 Full-Year Financial Guidance (in millions, except % and EPS amounts) (Updated) BioMarin will host a conference call and webcast to discuss third quarter 2024 financial results today, Tuesday, October 29, 2024, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at . About BioMarin BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin pursues treatments that offer new possibilities for patients and families around the world navigating rare or difficult to treat genetic conditions. To learn more, please visit .   Forward-Looking Statements This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Operating Margin percentage, and Non-GAAP Diluted EPS for the full-year 2024 and the underlying drivers of those results, such as the revenue opportunity represented by treatments for skeletal conditions, as well as the expectations relating to operating cash flow per year starting in 2027 and BioMarin's compound annual growth rate (CAGR) for total revenues through 2034; BioMarin's new corporate strategy and belief that such strategy will build on BioMarin's legacy of innovation to deliver even greater value to the patients it serves around the world and position BioMarin for significant growth; BioMarin's commitment to expand profitability while investing in innovation; BioMarin's ability to accelerate the VOXZOGO opportunity; the continued growth potential of BioMarin's Enzyme Therapies portfolio; the anticipated benefits from its organizational redesign efforts; BioMarin's updated strategy for ROCTAVIAN and its anticipated benefits; the timing of orders for commercial products; BioMarin's ability to meet product demand; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials; the clinical development and commercialization of BioMarin's product candidates and commercial products, including (i) the potential to leverage VOXZOGO in conditions beyond achondroplasia, such as hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, SHOX deficiency and other genetic short stature pathway conditions, (ii) the expected completion of enrollment for the pivotal study of VOXZOGO in hypochondroplasia in the first half of 2025, (iii) the expected expansion of VOXZOGO in the U.S. and BioMarin's ability to enable VOXZOGO access into more than 20 additional countries by 2027, (iv) BioMarin's expectation to initiate the first-in-human study for BMN 333 in early 2025, (v) BioMarin's expectation to receive initial proof-of-concept data regarding BMN 351 in 2025, (vi) BioMarin's expectation to start dosing the multiple-ascending dose phase of the study for BMN 349 by the end of the year, (vii) BioMarin's expectation to receive data from the phase 3 study with PALYNZIQ in adolescents ages 12-17 in 2025 to support a potential U.S. Supplemental Biologics License Application in the second half of the year, (viii) BioMarin's expectations to submit an IND for BMN 390 in the second half of 2025, and (ix) BioMarin's expectation to potentially submit an IND for BMN 370 during the second half of 2025; the expected benefits and availability of BioMarin's commercial products and product candidates; and potential growth opportunities and trends, including BioMarin's plans to drive growth of its Enzyme Therapies across the 80 countries in which they are available. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products; impacts of macroeconomic and other external factors on BioMarin's operations; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; actual sales of BioMarin's commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2024 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin®, BRINEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, ROCTAVIAN®, VIMIZIM® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners. Non-GAAP Information The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company's equity plans and convertible debt in periods when they are dilutive under Non-GAAP. The company's presentation of percentage changes in total revenues at Constant Currency rates, which is computed using current period local currency sales at the prior period's foreign exchange rates, is also a Non-GAAP financial measure. This measure provides information about growth (or declines) in the company's total revenue as if foreign currency exchange rates had not changed between the prior period and the current period. BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. Because Non-GAAP Income, Non-GAAP Operating Margin percentage, Non-GAAP Diluted EPS, Non-GAAP Weighted-Average Diluted Shares Outstanding and Constant Currency are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Non-GAAP Income and its components are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information: SOURCE BioMarin Pharmaceutical Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Results from Ongoing CANOPY Clinical Program for VOXZOGO Showcase Impact of C-Type Natriuretic Peptide (CNP) as Master Regulator of Growth-Related Development Across Multiple Genetic Skeletal Conditions New Data Highlight Meaningful Improvements in Health-Related Quality of Life for Children with Achondroplasia Investigator-Led Study Shows Significant Increases in Bone Length While Maintaining Bone Strength in Children with Achondroplasia Investigator-Led Study Observes Sustained Growth Gains in Children with Hypochondroplasia, Noonan Syndrome and Genetic Variants Associated with Idiopathic Short Stature SAN RAFAEL, Calif., Sept. 18, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that positive data from the CANOPY clinical program evaluating VOXZOGO® (vosoritide) in children with achondroplasia and other genetic skeletal conditions will be presented at the 16th International Skeletal Dysplasia Society meeting (ISDS) in Madrid, Sept. 18-21, 2024. These results include data showing that children with achondroplasia treated with VOXZOGO experienced meaningful improvements beyond height, such as in health-related quality of life (HRQoL), and increased bone length while maintaining bone strength. Researchers will also present encouraging data from ongoing investigator-led studies investigating treatment in children with other genetic skeletal conditions, including hypochondroplasia and Noonan syndrome, as well as those with genetic variants often associated with idiopathic short stature such as aggrecan (ACAN) deficiency and heterozygous NPR2 mutations. "VOXZOGO is now becoming the standard of care in achondroplasia, based on its proven effects on growth velocity, its safety profile and the clinical data demonstrating positive impact on proportionality and quality of life in treated children with achondroplasia," said Ravi Savarirayan, M.D., Ph.D., group leader of Skeletal Biology and Disease at Murdoch Children's Research Institute in Melbourne, Australia. Positive Impacts in Achondroplasia on Health Outcomes Including HRQoL and Maintenance of Bone Strength Qualitative insights elucidating caregiver perspectives of children with achondroplasia revealed that VOXZOGO had positive impacts on HRQoL across several physical, emotional and social functioning domains. Notable physical functioning improvements reported included improved motor skills (e.g., higher reach, walking, running, balance and cycling) and self-care, which are particularly meaningful outcomes for children and families impacted by the condition. Psychosocial functioning outcomes included improved confidence and social interactions. Additional results from an investigator-led analysis of BioMarin's Phase 2 111-205 study, previously shared at the 2024 International Conference on Children's Bone Health, demonstrated that children who received VOXZOGO (n=30) had significant increases in bone length and metacarpal cortical area after approximately five years of therapy, suggesting that treatment allowed the bone to remain strong as it lengthened. "VOXZOGO is the first and only approved treatment for children with achondroplasia, providing families with an option that can be initiated in infants, and we are excited to continue investigating its possibilities in other genetic skeletal conditions through our CANOPY clinical program," said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. "The data at ISDS continue to demonstrate the safety and efficacy of VOXZOGO, underscoring the clinical value of our approach with CNP as an effective, central regulator of growth-related development as our research seeks to transform the treatment landscape for families impacted by achondroplasia and other genetic skeletal conditions." Investigator-Led Studies of VOXZOGO Show Promising Efficacy and Safety Data in Hypochondroplasia and Other Genetic Skeletal Conditions Additional presentations at ISDS include efficacy data for VOXZOGO in ongoing research across genetic skeletal conditions beyond achondroplasia. Safety results were shown to be consistent with the well-characterized safety profile of VOXZOGO in achondroplasia. Results from an investigator-sponsored study showed sustained improvement in mean annualized growth velocity (AGV) and height standard deviation (SD) in 24 children with various genetic skeletal conditions, including Noonan syndrome and those with genetic variants associated with idiopathic short stature (ACAN deficiency and heterozygous NPR2 mutations), over one year of treatment. Updated investigator-sponsored results from the first clinical study of VOXZOGO for children with hypochondroplasia showed sustained improvement in AGV and hypochondroplasia-specific height SDs in 26 participants over one year. Treatment efficacy was similar to what had been previously seen in achondroplasia, and there were no new safety signals observed. Key presentations at ISDS are listed below, with all times in Central European Summer Time (CEST): Oral Presentations Adjuvant Therapeutic Enhances Bone Growth and Quality in Growing Mice with Moderate-to-Severe Osteogenesis Imperfecta: Exploration of a CNP Analog Oral #C-0012 Thursday, Sept. 19, 2:05 – 2:20 p.m. Vosoritide Increases Growth in Children with Hypochondroplasia: Phase 2 Trial Results Oral #C-0017 Friday, Sept. 20, 2:20 – 2:35 p.m. Vosoritide Improves Growth in RASopathies, ACAN and NPR2 Deficiency: Preliminary Data from a Phase 2 Trial Oral #C-0022 Friday, Sept. 20, 2:35 – 2:50 p.m. Body Composition by Bioelectrical Impedance Analysis (BIA) in Adults with Achondroplasia in CLARITY (the Achondroplasia Natural History Study) Oral #C-0027 Saturday, Sept. 21, 9:45 – 10 a.m. Poster Presentations Caregiver Perspectives on Vosoritide Treatment of Children with Achondroplasia Poster #C-0090 Thursday, Sept. 19, 3:50 – 4:50 p.m. Examining the Effect of Vosoritide Treatment on Bone Strength in Children with Achondroplasia Poster #C-0033 Thursday, Sept. 19, 3:50 – 4:50 p.m. The Achondroplasia Roadmap Poster #C-0066 Thursday, Sept. 19, 3:50 – 4:50 p.m. Design and Objectives of the Acorn Study: A Non-Interventional Study Evaluating Long-Term Safety in Achondroplasia Children Treated with Vosoritide Poster #C-0110 Friday, Sept. 20, 11 a.m. – 12 p.m. About the VOXZOGO CANOPY Clinical Program The CANOPY clinical program was designed to evaluate the potential of VOXZOGO (vosoritide) in children with various genetic skeletal conditions, including achondroplasia, hypochondroplasia, Noonan syndrome, SHOX deficiency, Turner syndrome and idiopathic short stature, with the goal of addressing the unmet needs of and expanding treatment options for children and families impacted by these conditions. Studies underway as part of the CANOPY program beyond achondroplasia include: CANOPY HCH-OS, a multinational observational study in children with hypochondroplasia. CANOPY HCH-3, a Phase 3 randomized, placebo-controlled, double-blind multicenter study in children with hypochondroplasia. CANOPY ISS-OS, a multinational observational study in children with idiopathic short stature. CANOPY ISS-2, a Phase 2 randomized, controlled, multicenter study in children with idiopathic short stature. CANOPY NS, TS, SHOX-D-2, a Phase 2 study in multiple genetic skeletal conditions including Noonan syndrome, Turner syndrome and SHOX deficiency. About VOXZOGO In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history. To date, approximately 3,500 people with achondroplasia around the world have received VOXZOGO. In total, VOXZOGO is available in 44 markets worldwide. Patient Support Accessing VOXZOGO To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail [email protected]. For more information about VOXZOGO, please visit . For additional information regarding this product, please contact BioMarin Medical Information at [email protected]. About Achondroplasia Achondroplasia is a rare genetic skeletal condition caused by a variation in the FGFR3 gene. It is characterized by disproportionate short stature and a potentially high burden of complications related to impaired endochondral bone growth. Approximately 80% of children with achondroplasia are born to parents of average stature as a result of a spontaneous variation in the FGFR3 gene. The worldwide incidence of achondroplasia is around one in 25,000 live births. VOXZOGO U.S. Important Safety Information What is VOXZOGO used for? VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO? VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO? The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken? VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO? Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Please see additional safety information in the full Prescribing Information and Patient Information. About BioMarin Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data at the 2024 International Skeletal Dysplasia Society meeting (ISDS), including the oral and poster presentations; the development of BioMarin's VOXZOGO program generally; VOXZOGO's efficacy, safety and impact on health-related quality of life (HRQoL) in children with achondroplasia, including impact on height, bone length, strength and HRQoL; the potential benefits of VOXZOGO for children with growth-related conditions beyond achondroplasia, including hypochondroplasia and Noonan syndrome, as well as those with genetic variants often associated with idiopathic short stature such as aggrecan (ACAN) deficiency and heterozygous NPR2 mutations; BioMarin's VOXZOGO CANOPY clinical program, including BioMarin's plans and expectations for clinical trials for hypochondroplasia, genetic variants associated with idiopathic short stature and other genetic skeletal conditions such as Noonan syndrome, Turner syndrome and SHOX deficiency; and BioMarin's ability to transform the treatment landscape for families impacted by achondroplasia and other genetic skeletal conditions. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned pre-clinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the Food and Drug Administration, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin®, BioMarin RareConnections® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc. SOURCE BioMarin Pharmaceutical Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

BioMarin’s headquarters in San Francisco iStock, Michael Vi BioMarin executives sought to calm an anxious investor base Wednesday with a public address and pledge to achieve a nearly 50% bump in annual revenue by 2027. But analysts were left wanting. BioMarin’s year has been defined by rounds of layoffs, an executive restructuring and months-long soul searching on the future of hemophilia gene therapy Roctavian. On Wednesday, executives finally revealed the long-awaited full picture of what they have been building towards. The company will restructure into three key units: skeletal conditions, enzyme therapies and Roctavian, all in an effort to unlock $4 billion in revenue by 2027. BioMarin expects two FDA approvals by that year—and 11 launches by 2034. Also within the next decade, the company said it will have products in Phase III development across seven indications. After 2027, the company expects mid-teen revenue growth to follow through 2034, with non-GAAP operating margins of 40% or more beginning in 2026 and $1.25 billion in operating cash flow in 2027 and beyond. BioMarin reaffirmed its revenue guidance of $2.75 billion to $2.85 billion for fiscal year 2024. CFO Brian Mueller said the $4 billion forecast does not include any mergers or acquisitions. Rather, one of the company’s existing products, Voxzogo, which was approved in 2021 for achondroplasia, will generate the bulk of the BioMarin’s forecasted $4 billion revenue. Specifically, the company plans to expand Voxzogo’s label in other growth disorders, following its October 2023 label expansion to achondroplasia patients of all ages with open growth platesin. The projection is also not highly dependent on Roctavian, the gene therapy approved in June 2023 but which has failed to successfully launch. As of its second quarter earnings release last month, Roctavian had treated just five patients and generated $7 million in sales total, far below the expected $100 million to $200 million from Roctavian in 2023 alone. BioMarin undertook a review of the program, putting a divestiture on the table as a possible option. But ultimately, the company decided to stick with it. Roctavian is the smallest business unit of the three outlined in the new plan. The company now expects to achieve profitability for the Roctavian business by the end of 2025 by reducing expenses to $60 million. During second quarter earnings, the company announced that sales of the gene therapy would be restricted to just the U.S., Germany and Italy, where it is reimbursed. Analyst reaction to BioMarin’s ambitious new plan was mixed, with some relieved and even impressed by the outlook, while others felt there was too much focus on the approved medicines and not enough on the pipeline as well as the potential competition. The pipeline discussion felt “cursory and left us wanting,” Truist Securities analysts wrote in a Thursday morning note. “What’s more critical for us and felt lacking during the event was strategy against key external events anticipated over the next 12-18 months that could impact the fate of BMRN more near-term and put post-2027 guidance at-risk.” CEO Alexander Hardy, just nine months into the top job, acknowledged in his opening remarks that investors have been clamoring for more detail on the company’s future, promising to answer all the questions that have been swirling since Roctavian came under the microscope. But he emphasized the most important metric to him in all this is the goal of expanding by four times the number of patients treated by BioMarin medicines by 2034. “I want to take this opportunity to reiterate again that our strong performance that we’re sharing today is really primarily based on our current products and our indications that we’re seeking, so our life cycle plans, and very little on molecules where we have not yet received the proof of concept data,” Hardy said. “And I think this is a really unique risk pro the industry that we’re able to commit to high levels of growth with very low levels of scientific risk in that growth outlook.” Many of the tough choices have already been implemented across BioMarin. Hardy said they have conducted a $500 million cost-reduction campaign, which included the deprioritzation of several early-stage assets. In the two weeks leading up to the investor day, BioMarin restructured its C-suite and laid off 225 people across global operations. In May, 170 employees lost their jobs . Doing ‘Good Deals Really Well’ Hardy said repeatedly on this week’s call that while his team did not have to execute a deal, he said BioMarin was certainly open to the possibility of external innovation—that is, M&A—and he laid out a specific roadmap of what the company wants. Hardy said the team will specifically be looking in the $1.5 billion range for assets or companies that fit into BioMarin’s “Core 5” values, which is used to assess internal innovation. Those values include genetically defined populations, targeted therapeutics, assessable endpoints, transformational impact and a commercially competitive profile. BioMarin has $1 billion in cash to deploy and Hardy said there are many cash constrained small cap biotechs out there to consider. “Very simply, I would say that we believe that there are assets out there that are worth more in our hands than where they’re sitting right now,” he said. Key to this hunt for deals will be James Sabry, who will become chief business officer as of October 7. Hardy said Sabry has a track record of “over 1,500 deals done.” That, he said, “should give investors confidence that we’re going to do good deals really well.” A deal could add a cherry on top of BioMarin’s forecast, Mueller explained. “Our revenue targets are not dependent upon business development. However, the right deals over time can be additive to what is already a substantial level of growth,” he said. “Also the right deals over time can have a multiplying effect, diversifying and augmenting our portfolio.” Mueller said that BioMarin will prioritize external innovation over returning capital to shareholders. The company and its board are regularly scanning opportunities for deals. “For the near term, we believe that investing in long-term, sustainable revenue growth has the potential to deliver more value to shareholders than the outright return of capital,” he said. Pipeline and Competition Get Short Shrift BioMarin’s investor day had been a long wait for investors and analysts, who were quick to speak out on the company’s new plan. “Operationally it’s impressive that under new management, BMRN can in just a few years attain competitive operating margins (>40%), driven by both expense discipline and better-than-expected revenues,” wrote Stifel’s analysts in a Wednesday afternoon note. The update was well above expectations, the firm said. Stifel added, however, that BioMarin did not specifically define how the clinical pipeline or Voxzogo competition from other companies will play in the guidance. Ascendis Pharma and BridgeBio have therapies in development for achondroplasia. BioMarin executives were confident that Voxzogo can face down the competition due to its market-leading position, patient experience and reluctance to switch treatments. Significant potential lies in hypochondroplasia, where BioMarin plans to target patients with the most severe disease, they said. Other label expansion opportunities include idiopathic short stature, Turner syndrome, Noonan syndrome and SHOX deficiency disorders. Meanwhile, investors will be keenly watching for a Phase III readout for Ascendis’ TransCon CNP for achondroplasia; the release is expected in the coming weeks. “This catalyst represents a swing factor for investors in their ability to get excited about the Vox bull case—and while TranCon’s a viable competitive threat (we actually believe more so than [BridgeBio’s] infigratinib at this stage), there’s clear trial risk here,” Stifel wrote. Jefferies analysts said they believe that BioMarin’s new outline does not change much for investors who were already negative about the stock. “If you didn’t like BMRN heading into investor day, will today’s event change your mind?” the firm wrote Wednesday afternoon. So far, Jefferies has heard “not really” from clients the analysts have spoken to. But Jefferies said that Hardy’s moves so far have been “rational and in line with what investors want to see from BioMarin.” Whether it will all go to plan remains to be seen, of course. “A lot can happen in a decade, particularly in a hyper-innovative space such as biotech,” Truist analysts wrote, “and we hesitate to give much credit to pipeline programs without anticipated visibility more near-term.”