Laronidase

The future of MPS I treatment is poised for significant advancements, driven by ongoing research and innovative therapies. Emerging therapies, such as stem cell gene therapy, IDUA Gene therapy, and Recombinant DNA, including OTL-203, Lepunafusp alfa (JR-171), RGX-111, and Iduronicrin genleukocel-T (ISP-001), offer novel approaches. These therapies have the potential to reshape the treatment landscape by improving patient outcomes and reducing the limitations of existing treatments. LAS VEGAS, May 7, 2025 /PRNewswire/ -- DelveInsight's Mucopolysaccharidosis Type I Market Insights report includes a comprehensive understanding of current treatment practices, Mucopolysaccharidosis type I emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Mucopolysaccharidosis Type I Market Report According to DelveInsight's analysis, the market size for Mucopolysaccharidosis type I was found to be USD 145 million in the 7MM in 2024. In 2024, the MPS I market size in the US was highest, accounting for 48% of the total market of 7MM. This figure is expected to grow significantly with the introduction of emerging therapies. According to DelveInsight's estimates, in 2024, there were approximately 660 diagnosed prevalent cases of MPS I in the 7MM. Of these, the United States accounted for 36% of the cases. Leading Mucopolysaccharidosis type I companies developing emerging therapies, such as Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, and others, are developing novel Mucopolysaccharidosis type I drugs that can be available in the Mucopolysaccharidosis type I market in the coming years. The promising Mucopolysaccharidosis type I therapies in the pipeline include OTL-203, Lepunafusp alfa (JR-171), RGX-111, Iduronicrin genleukocel-T (ISP-001), and others. In March 2025, REGENXBIO Inc. finalized its previously announced strategic partnership with Nippon Shinyaku. Under the agreement, REGENXBIO and Nippon Shinyaku will collaborate to develop and commercialize RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome, in the United States and Asia. In February 2025, at the 21st Annual WORLDSymposium, Orchard Therapeutics presented updated findings from a proof-of-concept study of OTL-203, an investigational gene therapy for the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), during an encore oral presentation. The presentation summarized key neurological, skeletal, and other clinical outcomes observed in the study. In January 2025, Immusoft announced positive results from the first engineered B Cell (ISP-001) in a human clinical trial, to be presented at the WORLDSymposium. Discover which therapies are expected to grab the major MPS I market share @ Mucopolysaccharidosis Type I Market Report Mucopolysaccharidosis Type I Overview MPS I is a rare inherited lysosomal storage disorder caused by a deficiency in the enzyme alpha-L-iduronidase (IDUA), which is essential for the breakdown of glycosaminoglycans (GAGs) such as dermatan sulfate and heparan sulfate. When these substances accumulate, they lead to progressive damage in cells and tissues, impacting various organ systems, including the skeletal, cardiovascular, respiratory, and nervous systems. The condition presents as a spectrum, with the most severe form being Hurler syndrome and the mildest form known as Scheie syndrome. Diagnosis of MPS I involves testing for elevated GAG levels, enzyme activity assays, and genetic analysis, all of which support early intervention, prognosis determination, and genetic counseling. Early detection is further aided by newborn screening programs. Ongoing disease management relies on comprehensive assessments such as pulmonary function tests, sleep studies, hearing evaluations, eye exams, imaging of the bones, and cognitive testing. Molecular diagnostics and early screening are critical to improving patient outcomes and guiding long-term care. Mucopolysaccharidosis Type I Epidemiology Segmentation The Mucopolysaccharidosis type I epidemiology section provides insights into the historical and current Mucopolysaccharidosis type I patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Mucopolysaccharidosis type I market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Diagnosed Prevalence of MPS I Severity-specific Diagnosed Prevalent Cases of MPS I Treated Cases of MPS I Hurler Syndrome Treatment Market The treatment landscape for MPS I (Hurler syndrome) is progressing gradually, with growing insights into both the disease's biology and its management. Traditional therapies like ERT and HSCT remain central, but advances in precision medicine and new pharmacological options are beginning to show promise. Despite these developments, a universal, comprehensive treatment approach remains elusive due to the disease's complexity and variability among patients, requiring a tailored, multidisciplinary strategy. Supportive care plays a critical role and includes various surgical procedures such as adenotonsillectomy, hernia repair, ventriculoperitoneal shunting, heart valve replacement, carpal tunnel release, and spinal decompression. Therapies like physical, occupational, and speech therapy, along with respiratory aids like CPAP, hearing devices, and medications for managing pain and gastrointestinal symptoms, are also essential. Vision issues may require corneal transplants, although surgical interventions in Hurler syndrome can be risky due to anesthetic complications. ALDURAZYME (laronidase), a recombinant version of the human IDUA enzyme produced using Chinese hamster ovary cells, was approved in the U.S., EU4, the UK (in 2003), and Japan (in 2006). It is indicated for patients with Hurler and Hurler-Scheie types of MPS I, and for symptomatic Scheie patients with moderate to severe manifestations. While ALDURAZYME carries a boxed warning due to potential safety concerns, it remains the only approved ERT for MPS I, with no biosimilars available despite the expiration of its patent. To know more about ALDURAZYME mechanism of action and laronidase cost, visit @ ALDURAZYME Patent Expiration Mucopolysaccharidosis Type I Pipeline Therapies and Key Companies OTL-203: Orchard Therapeutics/Kyowa Kirin Lepunafusp alfa (JR-171): JCR Pharmaceuticals RGX-111: REGENXBIO/Nippon Shinyaku Iduronicrin genleukocel-T (ISP-001): IMMUSOFT Discover more about MPS I drugs in development @ Mucopolysaccharidosis I Clinical Trials Market Mucopolysaccharidosis Type I Market Dynamics The MPS I market dynamics are expected to change in the coming years. Current treatments for MPS I, including ERT and hematopoietic stem cell transplantation, have demonstrated effectiveness in managing symptoms and improving the quality of life for patients with severe forms of the disease. Advances in genetic testing and the expansion of newborn screening programs have enabled earlier diagnosis, allowing for timely intervention and significantly improved patient outcomes. Increased awareness of MPS I, coupled with enhanced genetic counseling, has further contributed to earlier detection and better disease management. Additionally, the growing interest in rare diseases has attracted increased funding and support for research, accelerating the development of new and potentially more effective treatments for MPS I. These combined efforts are shaping a more promising future for patients and families affected by this rare lysosomal storage disorder. Furthermore, potential therapies are being investigated for the treatment of MPS I, and it is safe to predict that the treatment space will significantly impact the mucopolysaccharidosis therapeutics market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the MPS I market in the 7MM. However, several factors may impede the growth of the MPS I market. ERTs for MPS I, such as intravenous iduronidase, are lifelong treatments typically administered every week. While ERT has shown effectiveness in alleviating many somatic symptoms, it is unable to cross the blood-brain barrier, rendering it ineffective in addressing the neurological manifestations associated with severe forms of MPS I. This represents a significant limitation in the current therapeutic approach. As an autosomal recessive disorder, MPS I requires both parents to be carriers, which complicates family planning and increases the likelihood of recurrence in future pregnancies. Despite the benefits of available treatments like ALDURAZYME, the financial burden associated with long-term therapy remains a challenge for many patients and families. Additionally, serious adverse reactions—including life-threatening anaphylaxis—have been reported in some patients during or up to three hours after ALDURAZYME infusion, underscoring the need for careful monitoring and consideration of risk-benefit balance in treatment planning. Moreover, MPS I treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the MPS I market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the MPS I market growth. Scope of the Mucopolysaccharidosis Type I Market Report Therapeutic Assessment: Mucopolysaccharidosis Type I current marketed and emerging therapies Mucopolysaccharidosis Type I Market Dynamics: Key Market Forecast Assumptions of Emerging Mucopolysaccharidosis Type I Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Mucopolysaccharidosis Type I Market Access and Reimbursement Download the report to understand which factors are driving MPS I market trends @ Mucopolysaccharidosis Type I Market Trends Table of Contents Related Reports Mucopolysaccharidosis Type I Pipeline Mucopolysaccharidosis Type I Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key MPS I companies, including Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, BioMarin Pharmaceutical, Sanofi, among others. Mucopolysaccharidosis Market Mucopolysaccharidosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS companies, including Takeda Pharmaceutical, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Sanofi , among others. Mucopolysaccharidosis Type II Market Mucopolysaccharidosis Type II Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS II companies, including Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Abeona Therapeutics , among others. Mucopolysaccharidosis Type III Market Mucopolysaccharidosis Type III Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS III companies, including Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Lysogene, Inventiva , among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

First Quarter 2025 Total Revenues of $745 million (+15% Y/Y and +17% at Constant Currency Y/Y) First Quarter 2025 GAAP Diluted Earnings Per Share (EPS) of $0.95 (+107% Y/Y) First Quarter 2025 Non-GAAP Diluted EPS of $1.13 (+59% Y/Y) Conference Call and Webcast Scheduled Today at 4:30 p.m. ET SAN RAFAEL, Calif., May 1, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the first quarter ended March 31, 2025. "During the first quarter, we saw continued high demand for our innovative medicines resulting in strong revenue growth and profitability," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Products in our pipeline also advanced according to plan. In April, we shared positive top-line results from the Phase 3 PALYNZIQ® study for the treatment of adolescents with phenylketonuria between the ages of 12 and 17. Also in April, we were pleased to conclude enrollment in the pivotal study in hypochondroplasia with VOXZOGO, keeping us on track to launch in 2027, should data be supportive." Mr. Hardy continued, "For the remainder of 2025, we look forward to continued momentum in our global expansion of VOXZOGO® for achondroplasia. Across our Enzyme Therapies, we plan to build upon strong PALYNZIQ performance in the quarter, as well as initiatives to drive uptake of our other therapies to reach an even greater number of patients around the world. In addition to our strong financial outlook, we expect to advance multiple new indications with VOXZOGO in our CANOPY clinical program, share early clinical results from both BMN 351 for Duchenne Muscular dystrophy and BMN 333, our long-acting C-type natriuretic peptide, as well as execute on our business development strategy. We are delivering strong growth and profitability while we continue to implement BioMarin's new strategy and operating model. We look forward to seeing the benefits of this transformation flow through our results in the coming quarters and beyond." First Quarter 2025 Financial Highlights Total Revenues for the first quarter of 2025 were $745 million, an increase of 15% compared to the same period in 2024, driven by strong 40% year-over-year VOXZOGO revenue growth from new patients initiating therapy across all regions. In the quarter, revenues from BioMarin's Enzyme Therapies (ALDURAZYME®, BRINEURA®, NAGLAZYME®, PALYNZIQ and VIMIZIM®) increased 8% compared to the first quarter of 2024, driven by a combination of increased patient demand and the timing of large government orders in all regions. The increase was partially offset by lower KUVAN® product revenues attributed to continued generic competition as a result of the loss of market exclusivity. GAAP Net Incom e increased by $97 million to $186 million in the first quarter of 2025 compared to the same period in 2024, an increase of 109%, primarily attributed to higher gross profit driven by the factors noted above. The increase was also attributed to lower operating expenses following the termination of certain early stage development programs following the company's 2024 strategic portfolio review and focused ROCTAVIAN strategy announced in the second half of 2024. These increases were partially offset by higher tax provision primarily due to increase in taxable income. Non-GAAP Income increased by $81 million to $221 million in the first quarter of 2025 compared to the same period in 2024, representing 58% growth. The increase in Non-GAAP Income was primarily due to the factors noted above. First Quarter 2025 Business Highlights Innovation Skeletal Conditions: In March 2025, BioMarin presented new data demonstrating favorable safety and strong adherence in real-world clinical practice with VOXZOGO in children with achondroplasia under the age of 3 years old at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Meeting. No treatment-related adverse events nor any dose interruptions were reported among 63 children followed for up to 23.7 months. These real-world findings further validate VOXZOGO's established safety profile and reinforce the therapeutic benefit seen in clinical studies. The study's safety results, including in infants as young as 1 month old, add to the growing body of evidence supporting early treatment initiation with VOXZOGO, consistent with new international treatment guidelines published in the journal Nature Reviews Endocrinology earlier this year. In April 2025, BioMarin completed enrollment in its pivotal Phase 3 study with VOXZOGO in hypochondroplasia and the company is on track to share topline data in 2026, with a potential launch in 2027. BioMarin plans to leverage its multiyear track record treating children with achondroplasia, a related condition, to raise awareness and treat children with hypochondroplasia across the globe. The CANOPY clinical program is continuing to advance VOXZOGO in additional new indications, including idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency. With BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP), the company enrolled multiple cohorts of healthy volunteers in its first-in-human study, with initial pharmacokinetic (PK) data expected by year-end. Detailed data from this study is expected to be presented at a scientific forum in the first half of 2026. Pre-clinical data with BMN 333 demonstrated sustained 100 pM concentrations for free CNP, representing an approximate 2-3 fold increase versus published data in an analogous pre-clinical model for other long-acting CNP analogs. Additionally, BioMarin recently met with FDA and reached agreement on an overall clinical development plan for BMN 333 in achondroplasia. Assuming the Phase 1 data are supportive, the company plans to initiate a registration-enabling study in 2026, supporting a previously disclosed target for 2030 approval. BioMarin plans to seek similar agreements with additional global regulators in the coming months. The company announced in April that its pivotal study with PALYNZIQ for the treatment of adolescents between the ages of 12 and 17 met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels. These data will support the planned submission of applications in the second half of 2025 to expand PALYNZIQ age eligibility in the United States and Europe. Other Clinical Pipeline Programs: BMN 351, BioMarin's next generation oligonucleotide for Duchenne Muscular Dystrophy, and BMN 349, an oral therapeutic for Alpha-1 antitrypsin deficiency (AATD)-associated liver disease, continue to advance. Initial data for BMN 351 is anticipated to be presented at a scientific congress in the second half of 2025 (including muscle dystrophin levels from the 6 mg/kg cohort after 25 weeks of dosing). During a recent strategic portfolio assessment of R&D programs, BioMarin determined that the evolving profile for BMN 370, a pre-clinical candidate for the treatment of von Willebrand disease, did not meet its threshold for further development and commercialization. The program has been discontinued and impacted employees have been redeployed within BioMarin. Growth Total VOXZOGO revenue in the first quarter increased 40% compared to the same period in 2024, representing continued strong global demand since its commercial launch in 2021. As of the end of the quarter, children with achondroplasia in 49 countries around the world were being treated with VOXZOGO. In the U.S., BioMarin is investing in focused initiatives to drive continued expansion. These efforts include increasing field personnel to broaden the prescriber base and adding awareness platforms to drive adoption of VOXZOGO treatment. This is expected to begin increasing the rate of U.S. expansion in the second half of the year. Outside of the U.S. (OUS), from where the majority of VOXZOGO revenue is generated, uneven ordering patterns, consistent with BioMarin's other brands, were observed. This OUS dynamic is expected to result in VOXZOGO full-year revenues being more weighted towards the second half of 2025. Enzyme Therapies revenues grew 8% in the first quarter Y/Y, driven by strong continued demand for PALYNZIQ. Strong PALYNZIQ performance as well as solid growth from BioMarin's other enzyme treatments are expected to continue throughout 2025. Value Commitment In the first quarter of 2025, BioMarin delivered strong results across the business. Total revenues for the first quarter grew 15% Y/Y. First quarter GAAP Operating Margin of 30.0% expanded 16.4 percentage points Y/Y while GAAP Diluted EPS of $0.95 increased 107% Y/Y. First quarter Non-GAAP Operating Margin of 35.7% expanded 11.9 percentage points Y/Y while Non-GAAP Diluted EPS of $1.13 increased 59% Y/Y. These measures of profitability increased at rates faster than revenue growth, representing the company's focus on operational efficiency. During the quarter, BioMarin continued to realize the benefits of cost transformation initiatives implemented in 2024, resulting in a decrease in GAAP and Non-GAAP R&D and SG&A expenses Y/Y. Throughout the remainder of 2025, BioMarin expects to increase investments in VOXZOGO indication expansion, clinical pipeline development, and commercialization initiatives supporting the company's Skeletal Conditions and Enzyme Therapies business units. The company generated operating cash flows totaling $174 million in first quarter 2025, an increase of 271% compared to first quarter 2024. Total cash and investments at the end of the first quarter were approximately $1.8 billion, and with anticipated increasing profitability, BioMarin is positioned to generate increasing operating cash flow into the future. Today, the company reaffirmed its previously communicated 2025 full-year financial guidance, which reflects the impact of tariffs that have already been enacted but does not reflect the impact of potential future pharmaceutical tariffs. BioMarin has immaterial exposure to U.S. tariffs for China, Mexico and Canada across its global supply chain operations and product sales. 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. 2025 Full-Year Financial Guidance (in millions, except % and EPS amounts) BioMarin will host a conference call and webcast to discuss first quarter 2025 financial results today, Thursday, May 1, 2025, 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, Non-GAAP Diluted EPS and Operating Cash Flow for, in certain instances, the full-year 2025 and future periods, as well as profitability growth in 2025, and the underlying drivers of those results, such as the revenue opportunity represented by treatments for Skeletal Conditions, namely VOXZOGO, the expected demand and continued growth of BioMarin's Enzyme Therapies portfolio, including PALYNZIQ, and the expectation regarding the full realization of the benefits of BioMarin's cost transformation program; plans regarding BioMarin's revamped corporate strategy and operating model in 2025 and beyond, including expected growth in the Skeletal Conditions business unit and execution of BioMarin's business development strategy, 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) expected advancements of pipeline candidates, including BMN 333, BMN 349 and BMN 351, the anticipated initial data read-out for BMN 351 in the second half of 2025, the expected data and data presentation for BMN 333 in the first half of 2026 and plans to initiate a registration-enabling study for BMN 333 in 2026, as well as plans to seek similar agreements with additional global regulators in the coming months; (ii) plans to submit applications to expand PALYNZIQ age eligibility for the treatment of adolescents with phenylketonuria between the ages of 12 and 17 in the U.S. and Europe in the second half of 2025; (iii) expected topline data from the pivotal study in hypochondroplasia in 2026 and launch in 2027; (iv) the expectations regarding global expansion of VOXZOGO for achondroplasia and expected increase in the rate of U.S. expansion in the second half of 2025; (v) plans to advance five new VOXZOGO indications with BioMarin's CANOPY clinical program; and (vi) plans to reach greater number of patients around the world across BioMarin's Enzyme Therapies; the expected benefits and availability of BioMarin's commercial products and product candidates; and potential growth opportunities and trends. 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, such as trade wars and potential future pharmaceutical tariffs; 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 U.S. 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 Annual Report on Form 10-K for the fiscal year ended December 31, 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 R&D expenses and Non-GAAP SG&A expenses are defined by the company as GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. 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 R&D expenses, Non-GAAP SG&A expenses, 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 financial measures 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