Management's Discussion and Analysis of Financial Condition and Results of Operations
The following Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A) is intended to help the reader understand our results of operations and financial condition. The MD&A is provided as a supplement to, and should be read in conjunction with, our audited Consolidated Financial Statements and the accompanying notes to the Consolidated Financial Statements and other disclosures included in this Annual Report on Form 10-K, including the disclosures under "Risk Factors" in Part I, Item 1Aof this Annual Report on Form 10-K. These risks and uncertainties could cause actual results to differ significantly from those projected in forward-looking statements contained in this report or implied by past results and trends. Forward-looking statements are statements that attempt to forecast or anticipate future developments in our business, financial condition or results of operations. See the section titled "Forward-Looking Statements" that appears at the beginning of this Annual Report on Form 10-K. These statements, like all statements in this report, speak only as of the date of this Annual Report on Form 10-K (unless another date is indicated), and, except as required by law, we undertake no obligation to update or revise these statements in light of future developments. Our Consolidated Financial Statements have been prepared in accordance with United States (U.S.) generally accepted accounting principles (GAAP) and are presented in U.S. Dollars (USD).
Overview
We are a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Our 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, we seek to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. A summary of our commercial products, as of December 31, 2025, is provided below:
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Commercial Products
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Indication
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VOXZOGO (vosoritide)
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Achondroplasia
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Enzyme Therapies:
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VIMIZIM (elosulfase alpha)
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Mucopolysaccharidosis (MPS) IVA
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NAGLAZYME (galsulfase)
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MPS VI
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PALYNZIQ (pegvaliase-pqpz)
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Phenylketonuria (PKU)
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ALDURAZYME (laronidase)
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MPS I
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BRINEURA (cerliponase alfa)
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Neuronal ceroid lipofuscinosis type 2 (CLN2)
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KUVAN (sapropterin dihydrochloride)
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PKU
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ROCTAVIAN (valoctocogene roxaparvovec)
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Severe Hemophilia A
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2025 Financial Highlights
Key components of our results of operations include the following:
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Twelve Months Ended December 31,
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2025
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2024
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2023
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Total revenues
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$
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3,221.3
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$
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2,853.9
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$
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2,419.2
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Cost of sales
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$
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717.4
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$
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580.2
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$
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532.1
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Research and development (R&D)
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$
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921.9
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$
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747.2
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$
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746.8
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Selling, general and administrative (SG&A)
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$
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1,153.0
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$
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1,009.0
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$
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892.4
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Provision for income taxes
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$
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133.6
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$
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114.9
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$
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20.9
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Net income
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$
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348.9
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$
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426.9
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$
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167.6
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See "Results of Operations" below for discussion of our results for the periods presented.
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
Uncertainty Relating to Macroeconomic Environment
Conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions, could impact our global revenue sources and our overall business operations. The extent and duration of such effects remain uncertain and difficult to predict. We are actively monitoring and managing our response and assessing actual and potential impacts to our operating results and financial condition, as well as developments in our business, which could further impact the developments, trends and expectations described below. See the risk factor, "Our business is affected by macroeconomic conditions." described in "Risk Factors" in Part I, Item 1Aof this Annual Report on Form 10-K.
Business Developments
In 2025, we achieved $3.2 billion in total revenues, including a significant contribution from our ongoing expansion of VOXZOGO, and we continued to grow our commercial business and advance our product candidate pipeline. We believe that the combination of our internal research programs and partnerships and acquisitions of external assets will allow us to continue to develop and commercialize innovative therapies for people with serious and life-threatening rare diseases and medical conditions. We periodically conduct strategic portfolio assessment of research and development programs to determine which we believe have the strongest combination of scientific merit, opportunity for commercial success and potential value creation for stockholders. Based on such strategic portfolio assessments, certain programs that do not meet its threshold for further development and commercialization could be discontinued.
In December 2025, we entered into a definitive agreement to acquire Amicus Therapeutics, Inc. (Amicus), a publicly traded, global, biotechnology company for $14.50 per share in an all-cash transaction for a total consideration of approximately $4.8 billion. The pending acquisition is expected to strengthen our commercial portfolio by adding two new therapies for the treatment of Fabry disease and late-onset Pompe disease. The transaction is expected to close in the second quarter of 2026, subject to regulatory clearances, approval by the stockholders of Amicus and other customary closing conditions. We intend to finance the transaction through a combination of cash on hand and approximately $3.7 billion of non-convertible debt financing.
In December 2025, we entered into a debt financing commitment letter (the Commitment Letter) and related fee letter with certain lenders, pursuant to which the lenders have committed to provide us with debt financing up to approximately $3.7 billion (the Bridge Commitment) in the form of a 364-day senior secured bridge loan facility (Bridge Facility) for the pending acquisition of Amicus. No amounts had been drawn or were outstanding under the Bridge Commitment as of December 31, 2025. In February 2026, we issued $850.0 million in aggregate principal amount of 5.5% senior unsecured notes due 2034 (the 2034 Notes), and the proceeds from the issuance were deposited into an escrow account that will be used to finance the pending acquisition of Amicus. In connection with the issuance of the 2034 Notes, the Bridge Commitment was reduced from approximately $3.7 billion to $2.8 billion. In place of the Bridge Facility, we also expect to enter into a senior secured term loan facility for approximately $2.8 billion in aggregate principal and a new $600.0 million senior secured revolving credit facility in 2026 that will be executed prior to or concurrently with the closing of the pending Amicus acquisition. See "Financial Condition, Liquidity and Capital Resources" below for additional information.
In October 2025, we announced our plan to pursue options to divest ROCTAVIAN, including exploring out-licensing opportunities. Subsequently in December 2025, we committed to a plan to voluntarily withdraw ROCTAVIAN from the market due to lower than previously anticipated commercial opportunities. In connection with this strategic decision, we recorded approximately $240.0 million of restructuring charges in 2025 comprised of an inventory write-off, impairment of long-lived assets, severance and other costs. See Note 19to our accompanying Consolidated Financial Statements for additional details.
In July 2025, we completed the acquisition of Inozyme Pharma, Inc. (Inozyme), a publicly traded clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics. The acquisition is intended to strengthen our enzyme therapies portfolio by adding a late-stage enzyme replacement therapy, BMN 401 (formerly INZ-701), for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency. We accounted for this transaction as an asset acquisition since the lead asset, BMN 401, represents substantially all of the fair value of the gross assets acquired. See Note 20to our accompanying Consolidated Financial Statements for additional information related to Inozyme acquisition.
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
Results of Operations
Net Product Revenues
Net Product Revenues consisted of the following:
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Twelve Months Ended
December 31,
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2025
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2024
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2023
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2025 vs. 2024
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2024 vs. 2023
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VOXZOGO
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$
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926.9
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$
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735.1
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$
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469.9
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$
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191.8
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$
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265.2
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Enzyme Therapies:
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VIMIZIM
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792.1
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739.8
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701.0
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52.3
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38.8
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NAGLAZYME
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485.4
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479.6
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420.3
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5.8
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59.3
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PALYNZIQ
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433.3
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355.0
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303.9
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78.3
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51.1
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ALDURAZYME
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208.5
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183.9
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131.2
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24.6
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52.7
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BRINEURA
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186.4
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169.1
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161.9
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17.3
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7.2
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KUVAN
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99.6
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120.9
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180.8
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(21.3)
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(59.9)
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ROCTAVIAN
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35.6
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26.0
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3.5
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9.6
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22.5
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Total net product revenues
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$
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3,167.8
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$
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2,809.4
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$
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2,372.5
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$
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358.4
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$
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436.9
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Net Product Revenues include revenues generated from our commercial products. In the U.S., our commercial products, except for PALYNZIQ and ALDURAZYME, are generally sold to specialty pharmacies or end users, such as hospitals, which act as retailers. PALYNZIQ is distributed in the U.S. through certain certified specialty pharmacies under the PALYNZIQ Risk Evaluation and Mitigation Strategy program, and ALDURAZYME is marketed worldwide by Sanofi. Outside the U.S., our commercial products are sold to authorized distributors or directly to government purchasers or hospitals, which act as the end users.
The increase in Net Product Revenues in 2025 as compared to 2024 was primarily attributed to the following:
•VOXZOGO: higher sales volume from new patients initiating therapy across all regions;
•PALYNZIQ: higher sales volume from new patients initiating therapy, primarily in the U.S.;
•VIMIZIM: higher sales volume due to timing of orders in countries that place large government orders, primarily from countries in the Middle East and Latin America;
•ALDURAZYME: higher sales volume due to timing of order fulfillment to Sanofi as we recognize ALDURAZYME revenues when the product is released and control is transferred to Sanofi; and
•BRINEURA: higher sales volume from new patients initiating therapy, primarily in the U.S. and Latin America.
These increases were partially offset by the following:
•KUVAN: lower product revenues attributed to increasing generic competition as a result of the loss of market exclusivity.
In certain countries, governments place large periodic orders for our products. We expect that the timing of these large government orders will continue to be inconsistent, which has created in the past and may continue to create significant period to period variation in our revenues.
With respect to VOXZOGO, see also the risk factor "Our success depends on our ability to manage our growth and execute our corporate strategy." in "Risk Factors" included in Part I, Item 1A of this Annual Report for additional information on risk factors that could impact our business and operations.
We face exposure to movements in foreign currency exchange rates, and use foreign currency exchange forward contracts to hedge a percentage of our foreign currency exposure, primarily the Euro. Certain currencies are not included in our hedging program, such as the Argentine Peso. With respect to the risks posed by fluctuations of both hedged and unhedged currencies against the USD, see the risk factor "Our international operations pose currency risks, which may adversely affect our
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
operating results and net income" in "Risk Factors" included in Part I, Item 1Aof this Annual Report for additional information. The following table shows our Net Product Revenues denominated in USD and foreign currencies:
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Twelve Months Ended
December 31,
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2025
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2024
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2023
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2025 vs. 2024
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2024 vs. 2023
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Sales denominated in USD
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$
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1,604.2
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$
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1,366.0
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$
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1,137.8
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$
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238.2
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$
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228.2
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Sales denominated in foreign currencies
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1,563.6
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1,443.4
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1,234.7
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120.2
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208.7
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Total net product revenues
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$
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3,167.8
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$
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2,809.4
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$
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2,372.5
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$
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358.4
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$
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436.9
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Twelve Months Ended
December 31,
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2025
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2024
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2023
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2025 vs. 2024
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2024 vs. 2023
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Unfavorable impact of foreign currency exchange rates on product sales denominated in currencies other than USD
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$
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(40.7)
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$
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(107.8)
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$
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(100.0)
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$
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67.1
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$
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(7.8)
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The unfavorable impact of foreign currency exchange rates on USD reported results in 2025 was primarily driven by weakening of the Argentine Peso, Brazilian Real and Mexican Peso, partially offset by strengthening of the Euro. The unfavorable impact of foreign currency exchange rates on USD reported results in 2024 was primarily driven by weakening of the Argentine Peso and the Japanese Yen.
See "Quantitative and Qualitative Disclosures about Market Risk" in Part II, Item 7Aof this Annual Report on Form 10-K and the risk factor "Our international operations pose currency risks, which may adversely affect our operating results and net income" in "Risk Factors" included in Part I, Item 1Aof this Annual Report for information on currency exchange rate risk related to our Net Product Revenues.
Cost of Sales and Gross Margin
Cost of Sales includes raw materials, personnel, facility and other costs associated with manufacturing our commercial products. These costs include production materials, production costs at our manufacturing facilities, third-party manufacturing costs, amortization of technology transfer intangible assets and internal and external final formulation and packaging costs. Cost of Sales also includes royalties payable to third parties based on sales of our products, idle plant costs and charges for inventory write downs.
The following table summarizes our Cost of Sales and gross margin:
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Twelve Months Ended
December 31,
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2025
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2024
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2023
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2025 vs. 2024
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2024 vs. 2023
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Total revenues
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$
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3,221.3
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$
|
2,853.9
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$
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2,419.2
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$
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367.4
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$
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434.7
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Cost of sales
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$
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717.4
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$
|
580.2
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$
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532.1
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$
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137.2
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$
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48.1
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Gross margin
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77.7
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%
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79.7
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%
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78.0
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%
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(2.0)
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%
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1.7
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%
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Cost of Sales increased in 2025 compared to 2024 primarily due to $119.2 million write-off of ROCTAVIAN inventory as a result of our strategic decision in the fourth quarter of 2025 to voluntarily withdraw ROCTAVIAN from the market. Gross margin decreased in 2025 compared to 2024 primarily due to ROCTAVIAN inventory write-off, partially offset by increased sales volume of higher-margin products within our Enzyme Therapies portfolio.
Research and Development
R&D expense includes costs associated with the research and development of product candidates and post-marketing research commitments related to our commercial products. R&D expense primarily includes preclinical and clinical studies, personnel and raw materials costs associated with manufacturing clinical product, quality control and assurance, other R&D activities, R&D facilities and regulatory costs.
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
We group all of our R&D activities and related expense into three categories: (i) Research and early pipeline, (ii) Later-stage clinical programs and (iii) Marketed products as follows:
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Category
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Description
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Research and early pipeline
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R&D expense incurred in activities substantially in support of early research through the completion of phase 2 clinical trials, including drug discovery, toxicology, pharmacokinetics and drug metabolism and process development.
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Later-stage clinical programs
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R&D expense incurred in or related to phase 3 clinical programs intended to result in registration of a new product or a new indication for an existing product primarily in the U.S. or the EU.
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Marketed products
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R&D expense incurred in support of our marketed products that are authorized to be sold primarily in the U.S. or the EU. Includes clinical trials designed to gather information on product safety (certain of which may be required by regulatory authorities) and their product characteristics after regulatory approval has been obtained, as well as the costs of obtaining regulatory approval of a product in a new market after approval in either the U.S. or EU has been obtained.
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We manage our R&D expense by identifying the R&D activities we anticipate will be performed during a given period and then prioritizing efforts based on scientific data, probability of successful development, market potential, available human and capital resources and other similar considerations. We continually review our product pipeline and the development status of product candidates and, as necessary, reallocate resources among the research and development portfolio that we believe will best support the future growth of our business.
We continuously evaluate the recoverability of costs associated with pre-launch or pre-qualification manufacturing activities, if any, and capitalize the costs incurred related to those activities if we determine that recoverability is probable and therefore future revenues are expected. If the related product candidate's marketing application is rejected by the applicable regulators and the likelihood of future revenues for a product candidate become uncertain, the related manufacturing costs are expensed as R&D expenses.
R&D expense consisted of the following:
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Twelve Months Ended
December 31,
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2025
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2024
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2023
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2025 vs. 2024
|
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2024 vs. 2023
|
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Research and early pipeline
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$
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383.7
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$
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434.0
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$
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393.1
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$
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(50.3)
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$
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40.9
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Later-stage clinical programs
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308.3
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27.6
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62.6
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280.7
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(35.0)
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Marketed products
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229.9
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285.6
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291.1
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(55.7)
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(5.5)
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Total R&D expense
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$
|
921.9
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$
|
747.2
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$
|
746.8
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|
|
$
|
174.7
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|
|
$
|
0.4
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R&D expense increased in 2025 compared to 2024 primarily due to higher spend on Later-stage clinical programs as a result of the $221.0 million In-Process Research and Development (IPR&D) charge following the Inozyme acquisition and continued progression of VOXZOGO for hypochondroplasia. These increases were partially offset by lower spend on Research and early pipeline due to discontinued programs and lower spend on Marketed products mainly related to ROCTAVIAN.
Selling, General and Administrative
Sales and marketing (S&M) expense primarily consists of employee-related expenses for our sales group, brand marketing, patient support groups and pre-commercialization expenses related to our product candidates. General and administrative (G&A) expense primarily consists of corporate support and other administrative expenses, including employee-related expenses.
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
SG&A expenses consisted of the following:
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|
Twelve Months Ended
December 31,
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|
|
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|
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2025
|
|
2024
|
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2023
|
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2025 vs. 2024
|
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2024 vs. 2023
|
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S&M expense
|
|
$
|
530.2
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|
|
$
|
476.7
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|
|
$
|
488.4
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|
|
$
|
53.5
|
|
|
$
|
(11.7)
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G&A expense
|
|
622.8
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|
|
532.3
|
|
|
404.0
|
|
|
90.5
|
|
|
128.3
|
|
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Total SG&A expense
|
|
$
|
1,153.0
|
|
|
$
|
1,009.0
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|
|
$
|
892.4
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|
|
$
|
144.0
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|
|
$
|
116.6
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S&M expenses by product were as follows:
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|
|
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|
Twelve Months Ended
December 31,
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|
|
|
|
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|
|
2025
|
|
2024
|
|
2023
|
|
2025 vs. 2024
|
|
2024 vs. 2023
|
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Enzyme Therapies
|
|
$
|
247.1
|
|
|
$
|
219.0
|
|
|
$
|
225.3
|
|
|
$
|
28.1
|
|
|
$
|
(6.3)
|
|
|
VOXZOGO
|
|
184.2
|
|
|
134.1
|
|
|
108.9
|
|
|
50.1
|
|
|
25.2
|
|
|
ROCTAVIAN
|
|
30.7
|
|
|
76.7
|
|
|
104.5
|
|
|
(46.0)
|
|
|
(27.8)
|
|
|
Other
|
|
68.2
|
|
|
46.9
|
|
|
49.7
|
|
|
21.3
|
|
|
(2.8)
|
|
|
Total S&M expense
|
|
$
|
530.2
|
|
|
$
|
476.7
|
|
|
$
|
488.4
|
|
|
$
|
53.5
|
|
|
$
|
(11.7)
|
|
The increase in S&M expense for 2025 compared to 2024 was primarily due to increased spending related to global expansion of VOXZOGO for achondroplasia and pre-launch activities on VOXZOGO for hypochondroplasia, and higher spend on demand generating activities for Enzyme Therapies. These increases in S&M spend were partially offset by reduced activities related to ROCTAVIAN as we focused on our commercial efforts in the U.S., Germany and Italy in 2025.
The increase in G&A expense for 2025 compared to 2024 was primarily due to $118.5 million of restructuring charges related to impairment of long-lived assets as a result of our strategic decision in the fourth quarter of 2025 to voluntarily withdraw ROCTAVIAN from the market, and partially due to incremental administrative costs related to ongoing support of business initiatives during the year. These increases were partially offset by bad debt expense recorded in the fourth quarter of 2024 that did not recur in 2025.
Intangible Asset Amortization and Gain on Sale of Nonfinancial Assets
Changes during the periods presented for Intangible Asset Amortization and Gain on Sale of Nonfinancial Assets were as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Twelve Months Ended
December 31,
|
|
|
|
|
|
|
2025
|
|
2024
|
|
2023
|
|
2025 vs. 2024
|
|
2024 vs. 2023
|
|
Amortization of intangible assets
|
$
|
19.4
|
|
|
$
|
43.3
|
|
|
$
|
62.2
|
|
|
$
|
(23.9)
|
|
|
$
|
(18.9)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Gain on sale of nonfinancial assets
|
$
|
-
|
|
|
$
|
10.0
|
|
|
$
|
-
|
|
|
$
|
(10.0)
|
|
|
$
|
10.0
|
|
Amortization of intangible assets: the decrease in amortization expense for 2025 as compared to 2024 was due to the increase in the estimated useful life of an intangible asset as a result of the extension of a patent during the second half of 2024 and an intangible asset becoming fully amortized during the fourth quarter of 2024.
Gain on Sale of Nonfinancial Assets: in the first quarter of 2024, we recognized a gain of $10.0 million due to a third party's achievement of a regulatory approval milestone related to previously sold intangible assets.
Interest Income
We invest our cash equivalents and investments in U.S. government securities and other high credit quality debt securities in order to limit default and market risk.
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Twelve Months Ended
December 31,
|
|
|
|
|
|
|
2025
|
|
2024
|
|
2023
|
|
2025 vs. 2024
|
|
2024 vs. 2023
|
|
Interest income
|
$
|
74.9
|
|
|
$
|
74.9
|
|
|
$
|
58.3
|
|
|
$
|
-
|
|
|
$
|
16.6
|
|
Interest Income during 2025 compared to 2024 was relatively flat. We expect Interest Income to decrease over the next 12 months due to lower cash and investment balances as the pending Amicus acquisition will be financed through a combination of cash on hand and non-convertible debt financing.
Interest Expense
We incur interest expense primarily on our convertible debt. Interest Expense for the periods presented was as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Twelve Months Ended
December 31,
|
|
|
|
|
|
|
2025
|
|
2024
|
|
2023
|
|
2025 vs. 2024
|
|
2024 vs. 2023
|
|
Interest expense
|
$
|
10.9
|
|
|
$
|
12.7
|
|
|
$
|
17.3
|
|
|
$
|
(1.8)
|
|
|
$
|
(4.6)
|
|
Interest Expense decreased in 2025 as compared to 2024 primarily due to settlement of 2024 Notes that matured in August 2024. We expect Interest Expense to increase over the next 12 months due to financing related to the pending Amicus acquisition, including the 2034 Notes issued on February 12, 2026. See Note 20to our accompanying Consolidated Financial Statements for additional information regarding financing related to the pending acquisition of Amicus.
Other Income (Expense), Net
Other Income (Expense), Net for the periods presented was as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Twelve Months Ended
December 31,
|
|
|
|
|
|
|
2025
|
|
2024
|
|
2023
|
|
2025 vs. 2024
|
|
2024 vs. 2023
|
|
Other income (expense), net
|
$
|
9.0
|
|
|
$
|
(4.7)
|
|
|
$
|
(38.2)
|
|
|
$
|
13.7
|
|
|
$
|
33.5
|
|
The increase in Other Income (Expense), Net, in 2025 compared to 2024 was primarily due to proceeds from insurance related to damaged goods.
Provision for Income Taxes
Provision for Income Taxes for the periods presented was as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Twelve Months Ended
December 31,
|
|
|
|
|
|
|
2025
|
|
2024
|
|
2023
|
|
2025 vs. 2024
|
|
2024 vs. 2023
|
|
Provision for income taxes
|
$
|
133.6
|
|
|
$
|
114.9
|
|
|
$
|
20.9
|
|
|
$
|
18.7
|
|
|
$
|
94.0
|
|
Our Provision for Income Taxes in 2025 and 2024 consisted of state, federal and foreign current tax expense which was offset by foreign tax credits, and deferred tax benefits from federal orphan drug credits and federal R&D credits. In July 2025, the One Big Beautiful Bill (OBBB Act) was signed into law in the U.S. This legislation includes a broad range of U.S. tax reforms provisions which become effective through 2027. Those effective in 2025 are reflected in our 2025 results. The Provision for Income Taxes in 2025 increased compared to 2024, primarily due to non-deductible acquired IPR&D related to the Inozyme acquisition, tax expense related to the expiration of unexercised options, partially offset by reduction in tax expense as a result of the OBBB Act.
Certain countries in which we have operations, including Ireland, have adopted Pillar Two framework, recently released from the Organisation for Economic Co-operation and Development (OECD), including a minimum tax rate of 15%. The U.S. has not enacted legislation to adopt the Pillar Two framework. The adoption of the Pillar Two framework did not have a material impact
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
on our effective tax rate and we plan to continue evaluating additional guidance released by the OECD, along with the pending legislative adoption by additional individual countries.
Results of Operations 2024 Compared to 2023
For a discussion of our results of operations pertaining to 2024 as compared to 2023 see Item 7, "Management's Discussion and Analysis of Financial Condition and Results of Operations" in our Annual Report on Form 10-K for the year ended December 31, 2024 (filed with the Securities and Exchange Commission (SEC) on February 24, 2025).
Financial Condition, Liquidity and Capital Resources
Our cash, cash equivalents, and investments as of December 31, 2025 and 2024 were as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
2025
|
|
2024
|
|
2025 vs. 2024
|
|
Cash and cash equivalents
|
$
|
1,311.7
|
|
|
$
|
942.8
|
|
|
$
|
368.9
|
|
|
Short-term investments
|
248.9
|
|
|
194.9
|
|
|
54.0
|
|
|
Long-term investments
|
492.2
|
|
|
521.2
|
|
|
(29.0)
|
|
|
Total cash, cash equivalents and investments
|
$
|
2,052.8
|
|
|
$
|
1,658.9
|
|
|
$
|
393.9
|
|
We believe cash generated from sales of our commercial products, in addition to our cash, cash equivalents and short-term investments, including proceeds from the 2034 Notes and external financings, will be sufficient to satisfy our liquidity requirements for at least the next 12 months, including our agreement to acquire Amicus in an all-cash transaction. We believe we will meet longer-term expected future cash requirements and obligations through a combination of cash flows from operating activities and available cash and long-term investment balances. We will need to raise additional funds by issuing equity, debt or convertible securities, taking loans or entering into collaborative or other agreements if we are unable to satisfy our liquidity requirements. For example, we may require additional financing to fund the repayment of our outstanding indebtedness, future milestone payments and our future operations, including the commercialization of our products and product candidates currently under development, preclinical studies and clinical trials, and potential licenses and acquisitions. The timing and mix of our funding alternatives could change depending on many factors, including how much we elect to spend on our development programs, potential licenses and acquisitions of complementary technologies, products and companies or if we settle our convertible debt in cash. In addition, depending on prevailing market conditions, our liquidity requirements, contractual restrictions, and other factors, we may also from time to time seek to retire or purchase our outstanding debt through cash purchases and/or exchanges for equity securities, in open market purchases, privately negotiated transactions or otherwise.
We are mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions could affect our ability to achieve our goals. In addition, we sell our products in certain countries that face economic volatility and weakness. Although we have historically collected receivables from customers in such countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to be unable to pay for our products. We will continue to monitor these conditions and will attempt to adjust our business processes, as appropriate, to mitigate macroeconomic risks to our business.
Our cash flows for each of the years ended December 31, 2025 and 2024 were as follows:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
2025
|
|
2024
|
|
2025 vs. 2024
|
|
Net cash provided by operating activities
|
$
|
828.0
|
|
|
$
|
572.8
|
|
|
$
|
255.2
|
|
|
Net cash provided by (used in) investing activities
|
$
|
(414.2)
|
|
|
$
|
136.5
|
|
|
$
|
(550.7)
|
|
|
Net cash used in financing activities
|
$
|
(42.4)
|
|
|
$
|
(526.4)
|
|
|
$
|
484.0
|
|
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
The increase in net cash provided by operating activities in 2025 compared to 2024 was attributed to increase in net income adjusted for non-cash items mainly related to inventory write-off and asset impairments for ROCTAVIAN and IPR&D charges from Inozyme acquisition, and partially due to the timing of payments to vendors and cash receipts from our customers.
The increase in net cash used in investing activities in 2025 compared to 2024 was primarily attributable to $285.2 million net cash paid for the acquisition of Inozyme, lower net maturities of available-for-sale securities, and higher purchases of property, plant and equipment.
The decrease in net cash used in financing activities in 2025 compared to 2024 was primarily due to $495.0 million settlement of the 2024 Notes that matured in August 2024. The decrease was also attributable to lower proceeds from exercises of equity awards in 2025.
Financing and Credit Facilities
In December 2025, in connection with the pending acquisition of Amicus, we entered into the Commitment Letter and related fee letter with certain lenders, pursuant to which the lenders have committed to the Bridge Commitment, to provide us with debt financing in an aggregate principal amount of up to approximately $3.7 billion in the form of the 364-day senior secured Bridge Facility, subject to customary conditions and entry into definitive financing and ancillary documentation as set forth therein. No amounts had been drawn or were outstanding under the Bridge Commitment as of December 31, 2025.
In February 2026, we issued $850.0 million in aggregate principal amount of the 2034 Notes, and the proceeds from the issuance were deposited into an escrow account that will be used to finance the pending acquisition of Amicus. In connection with the issuance of the 2034 Notes, the Bridge Commitment was reduced from approximately $3.7 billion to $2.8 billion. In the event that the acquisition is not completed on or prior to December 19, 2026, or upon the occurrence of certain other events, we will be required to redeem all of the 2034 Notes at par and pay any accrued and unpaid interest. In place of the Bridge Facility, we also expect to enter into a senior secured term loan facility for approximately $2.8 billion in aggregate principal and a new $600.0 million senior secured revolving credit facility in 2026 (the New Revolving Facility) that will be executed prior to or concurrently with the closing of the pending Amicus acquisition. Upon entry into the senior secured term loan facility, the remaining Bridge Commitment will be reduced to zero. Under the New Revolving Facility, we may also borrow up to $150 million to pay fees and expenses related to the pending acquisition of Amicus.
Our $600.0 million (undiscounted) of total convertible debt as of December 31, 2025 consisting of our 1.25% senior subordinated convertible notes due in 2027 (the 2027 Notes), will impact our liquidity due to semi-annual cash interest payments and repayment of the principal amount in cash at maturity in May 2027 if not converted.
Our $600.0 million unsecured revolving credit facility (Revolving Facility) as of December 31, 2025 is intended to finance ongoing working capital needs and other general corporate initiatives. The Revolving Facility matures in August 2029 and contains financial covenants including a maximum total net leverage ratio and a minimum interest coverage ratio. As of December 31, 2025 there were no amounts outstanding under the Revolving Facility and we were in compliance with all covenants. The New Revolving Facility is expected to replace the existing Revolving Facility.
See Note 10to our accompanying Consolidated Financial Statements for additional discussion on our convertible debt and credit facility.
Material Cash Requirements
Purchase and Lease Obligations
As of December 31, 2025, we had purchase obligations of approximately $590.8 million, of which $354.1 million is expected to be paid in 2026. Our purchase obligations are primarily related to firm purchase commitments entered into in the normal course of business to procure active pharmaceutical ingredients, certain inventory-related items, certain third-party R&D services, production services and facility construction services.
As of December 31, 2025, we had lease payment obligations of $57.3 million, of which $10.3 million is payable in 2026. See Note 9to our accompanying Consolidated Financial Statements for details on our lease liabilities.
Unrecognized Tax Benefits
As of December 31, 2025, our liability for unrecognized tax benefits was $380.9 million. Due to their nature, we cannot reasonably estimate the timing of future payments. See Note 15to our accompanying Consolidated Financial Statements for a full discussion on our income taxes.
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
Critical Accounting Estimates
In preparing our Consolidated Financial Statements in accordance with U.S. GAAP and pursuant to the rules and regulations promulgated by the SEC, we make assumptions, judgments and estimates that can have a significant impact on our net income/loss and affect the reported amounts of certain assets, liabilities, revenue and expenses, and related disclosures. On an ongoing basis, we evaluate our estimates and discuss our critical accounting policies and estimates with the Audit Committee of our Board of Directors. We base our estimates on historical experience and various other assumptions that we believe to be reasonable under the circumstances. Actual results could differ materially from these estimates under different assumptions or conditions. Historically, our assumptions, judgments and estimates relative to our critical accounting policies have not differed materially from actual results.
Our significant accounting policies are described in Note 1to our accompanying Consolidated Financial Statements included in this Annual Report on Form 10-K. We believe the critical accounting estimates below reflect the most critical judgments and estimates used in the preparation of our Consolidated Financial Statements.
Revenue Recognition and Related Allowances
Net Product Revenues - We recognize revenue when the customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services. For ALDURAZYME revenues, we receive a payment ranging from 39.5% to 50% on worldwide net ALDURAZYME sales by Sanofi depending on sales volume, which is included in Net Product Revenues in our Consolidated Statements of Income. We recognize our best estimate of the entire revenue that we expect to receive when the product is released and control is transferred to Sanofi. We record ALDURAZYME net product revenues based on the estimated variable consideration payable when the product is sold through by Sanofi. Differences between the estimated variable consideration to be received and actual payments received are not expected to be material. If actual results vary from our estimates, we will make adjustments, which would affect Net Product Revenues and earnings in the period such variances become known.
Gross-to-Net Sales Adjustments - We record product sales net of estimated mandatory and supplemental discounts to government payers, discounts to private payers and other related charges. Rebates, cash discounts and distributor fees represent the majority of our gross-to-net deductions and are recorded in the same period the related sales occur. Rebates may include amounts paid to Medicaid or other U.S. or foreign government programs, certain managed care providers, or other payers. Rebates, branded co-pay assistance programs, cash discounts and distributor fees are estimates based on contractual arrangements or statutory obligations, which may vary by product and payer. Estimation requires evaluation of our actual historical experience, customer and payer mix, current contractual and statutory obligations, patient outcomes, specific known market events and trends and industry data. We evaluate our customer and payer mix to estimate which sales will be subject to these revenue dilutive items and consider changes to government program guidelines or contractual obligations that would impact the actual rebates and/or our estimates of which sales qualify for such rebates. Any necessary adjustments to our reserves are made each quarter to reflect current information. We believe the methodologies that we use to estimate allowances are reasonable and appropriate given the facts and circumstances. However, actual results may differ significantly from our estimates.
The following table summarizes the consolidated activities and ending balances of all our gross-to-net sales adjustments:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at Beginning of Year
|
|
Provision for Current Period
Sales
|
|
Payments
|
|
Balance at End of Year
|
|
Year ended December 31, 2025
|
|
$
|
195.0
|
|
|
$
|
487.8
|
|
|
$
|
(436.7)
|
|
|
$
|
246.1
|
|
|
Year ended December 31, 2024
|
|
$
|
152.1
|
|
|
$
|
435.1
|
|
|
$
|
(392.2)
|
|
|
$
|
195.0
|
|
|
Year ended December 31, 2023
|
|
$
|
115.0
|
|
|
$
|
370.7
|
|
|
$
|
(333.6)
|
|
|
$
|
152.1
|
|
Income Taxes
We calculate and provide for income taxes in each of the tax jurisdictions in which we operate. Our Consolidated Balance Sheets reflect net deferred tax assets and liabilities, which are measured using enacted tax rates. The net deferred tax assets primarily represent the tax benefit of tax credits and timing differences between book and tax recognition of certain revenue and expense items, net of a valuation allowance. When it is more likely than not that all or some portion of deferred tax assets may not be realized, we establish a valuation allowance for the amount that may not be realized. We utilize financial projections to support our net deferred tax assets, which contain significant assumptions and estimates of future operations. If such assumptions were to differ significantly, it may have a material impact on our ability to realize our net deferred tax assets. Changes in our valuation allowance will result in a change to tax expense.
Management's Discussion and Analysis of Financial Condition and Results of Operations (continued)
(In millions of U.S. Dollars, except as otherwise disclosed)
We establish liabilities or reduce assets for certain tax positions when we believe those certain tax positions are not more likely than not to be sustained if challenged. Each quarter, we evaluate these uncertain tax positions and adjust the related tax assets and liabilities in light of changing facts and circumstances.
We are subject to income taxes in the U.S. and various foreign jurisdictions, including Ireland. Due to economic and political conditions, various countries are actively considering changes to existing tax laws. We cannot predict the form or timing of potential legislative changes that could have a material adverse impact on our results of operations. Management is not aware of any potential changes that would have a material effect on our Consolidated Financial Statements. See Note 15to our accompanying Consolidated Financial Statements for additional discussion.
Valuation of assets and liabilities in connection with acquisitions
We have acquired and continue to acquire intangible assets in connection with business combinations and asset acquisitions. These intangible assets consists primarily of IPR&D for product candidates. Discounted cash flow models are typically used to determine the fair value of acquired intangible assets for the purposes of allocating consideration paid to the net assets in an acquisition. These models require us to make certain judgments, which include:
•developing appropriate probability of success rates for unapproved product candidates considering their stages of development;
•estimating time and resources needed to complete the development and approval of the product;
•projecting time to approval;
•risks related to the viability of potential alternative treatments in future target markets;
•revenue projections;
•and discount rate.
The judgments made in determining estimated fair values assigned to assets acquired and liabilities assumed in an acquisition, as well as estimated asset lives, can materially affect our consolidated results of operations. The fair values of intangible assets, including acquired IPR&D, are determined using information available near the acquisition date based on estimates and assumptions that are deemed reasonable by management. Depending on the facts and circumstances, we may deem it necessary to engage an independent valuation expert to assist in valuing significant assets and liabilities. The fair values of identifiable intangible assets are primarily determined using the income method.
Impairments of Long-Lived Assets
We assess changes in economic, regulatory and legal conditions and make assumptions regarding estimated future cash flows in evaluating the value of our property, plant and equipment, goodwill and other long-lived assets. We periodically evaluate whether current facts or circumstances indicate that the carrying values of our long-lived assets may not be recoverable. Should there be an indication of impairment, we test for recoverability by comparing the estimated undiscounted future cash flows expected to result from the use of the asset or asset group and its eventual disposition to the carrying amount of the asset or asset group. Any excess of the carrying value of the asset or asset group over its estimated fair value is recognized as an impairment loss.
Recent Accounting Pronouncements
See Note 1to our accompanying Consolidated Financial Statements for a full description of recent accounting pronouncements and our expectation of their impact on our results of operations and financial condition.