AMICUS THERAPEUTICS, INC.

Amicus Therapeutics, Inc. is a global biotechnology company focused on developing and delivering transformative medicines for people living with rare diseases. The company operates in the rare disease pharmaceutical and biotechnology industry, which is characterized by rapidly advancing technologies, intense competition, and a strong emphasis on proprietary products. Key structural forces shaping competition include the significant financial resources and expertise of larger competitors, the emergence of generic and biosimilar products, and the regulatory environment governing orphan drug approvals and exclusivity. Amicus sits within this landscape as a leading company with two marketed therapies for Fabry disease and Pompe disease, and a pipeline candidate for Focal Segmental Glomerulosclerosis (FSGS).

Amicus faces substantial competition from major pharmaceutical and biotechnology companies. For Fabry disease, competitors include Sanofi (Fabrazyme), Takeda (Replagal), and Chiesi (ELFABRIO), as well as Idorsia (lucerastat) and Sangamo (isaralgagene civaparvovec) in development. For Pompe disease, competitors include Sanofi (Myozyme/Lumizyme, Nexviazyme/Nexviadyme), Bayer (ACTUS-101), and Maze/Shionogi (MZE001). For FSGS, competitors include Travere (sparsentan) and Vertex (VX-147). The company's stated competitive advantages include its oral precision medicine Galafold for Fabry disease, which targets patients with amenable genetic variants, and its novel two-component treatment Pombiliti + Opfolda for Pompe disease, designed to enhance cellular uptake of the enzyme. Amicus also holds orphan drug designations and patents that provide market exclusivity.

Amicus generates revenue through the sale of its two marketed therapies: Galafold (migalastat HCl) for Fabry disease and Pombiliti + Opfolda (cipaglucosidase alfa-atga/miglustat) for Pompe disease. Revenue is recognized at a point in time when pharmacies or distributors obtain control of the products. The transaction price is determined based on fixed consideration net of estimates for variable consideration, which primarily consists of discounts and rebates due to foreign and U.S. government programs. The company's primary customer segments are government authorities and other third-party payors, with orders generally received from distributors and pharmacies. The company also has a pipeline of product candidates, including DMX-200 for FSGS, which it has licensed for U.S. commercialization rights.

Galafold is an oral precision medicine approved in over 40 countries, including the U.S., E.U., U.K., and Japan, for the treatment of adults with Fabry disease who have amenable genetic variants. It is designed to bind to and stabilize an endogenous alpha-galactosidase A enzyme. For the year ended December 31, 2025, Galafold revenue was $521.7 million ¹, representing an increase of $63.6 million compared to the prior year. This revenue was split between U.S. sales of $215.8 million ² and ex-U.S. sales of $305.9 million ³. Galafold has been granted orphan drug designation in the U.S., E.U., U.K., Japan, and several other countries.

Pombiliti + Opfolda is a novel two-component treatment for adults living with late-onset Pompe disease. Pombiliti is a uniquely engineered recombinant human acid alpha-glucosidase enzyme administered intravenously, and Opfolda is an orally administered stabilizer. The combination is approved in the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan. For the year ended December 31, 2025, Pombiliti + Opfolda revenue was $112.5 million ⁴, representing an increase of $42.3 million compared to the prior year. This revenue was split between U.S. sales of $48.3 million ⁵ and ex-U.S. sales of $64.2 million ⁶. The product has been granted orphan drug designation or status in the U.S., U.K., Switzerland, and Japan, and data exclusivity in the E.U. Additionally, the company has a pipeline candidate, DMX-200, a small molecule inhibitor of the chemokine receptor 2 under development in a pivotal Phase 3 study (ACTION3) for the treatment of FSGS kidney disease. DMX-200 has orphan drug designation from the FDA for the treatment of FSGS.

On April 30, 2025, Amicus entered into an exclusive license agreement with Dimerix Bioscience Pty Limited for the U.S. commercialization rights of Dimerix' Phase 3 drug candidate, DMX-200, for treatment of FSGS and other indications. Amicus paid Dimerix an upfront payment of $30 million ⁷. On December 19, 2025, Amicus entered into a definitive merger agreement with BioMarin Pharmaceutical Inc. Under the terms, all shares of Amicus common stock outstanding will be cancelled and converted into the right to receive $14.50 per share in cash ⁸. The transaction is expected to close in the second quarter of 2026. In October 2024, Amicus entered into a non-exclusive, royalty-free license with Teva to market a generic version of Galafold in the U.S. beginning on January 30, 2037 ⁹, or earlier in certain circumstances. In December 2025, Amicus resolved patent litigation with Aurobindo and Lupin on substantially similar terms, allowing them to market generic versions of Galafold in the U.S. beginning on January 30, 2037 ¹⁰, or earlier in certain circumstances.

For the year ended December 31, 2025, total net product sales were $634.2 million ¹¹, compared to $528.3 million ¹² in 2024 and $399.4 million ¹³ in 2023. Gross profit was $561.3 million ¹⁴ in 2025, compared to $475.4 million ¹⁵ in 2024. The company reported a net loss attributable to common stockholders of $27.1 million ¹⁶ for 2025, compared to a net loss of $56.1 million ¹⁷ in 2024 and $151.6 million ¹⁸ in 2023. The improvement in net loss was driven by revenue growth and a $19.7 million favorable variance in other expense/income primarily related to foreign exchange rate movements. Total cash, cash equivalents, and marketable securities as of December 31, 2025 were $293.5 million ¹⁹.

A key growth vector is the continued commercial expansion of Galafold in Fabry disease. The company plans to continue to launch Galafold in additional countries upon receipt of marketing authorization. The company believes that approximately 35-50% of the Fabry disease patient population may benefit from treatment with Galafold as a monotherapy. Increased awareness of Fabry disease, newborn screening in several U.S. states, family screening, and access to lower cost genetic testing are contributing to the continued expansion of Fabry diagnosis.

Another major growth vector is the commercial and regulatory success of Pombiliti + Opfolda in Pompe disease. The product has been approved in the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Clinical studies are ongoing in pediatric patients for both the late-onset Pompe disease and infantile-onset Pompe disease populations. Additionally, the company has licensed exclusive rights to commercialize DMX-200 in the U.S. for treatment of FSGS, which is in a pivotal Phase 3 study (ACTION3). Dimerix announced completion of enrollment for ACTION3 (n=286) ²⁰ in December 2025.

The filing does not provide specific margin trajectory, cost structure evolution, or efficiency targets.

The company relies on contract manufacturers to supply active biopharmaceutical ingredients and finished goods. Pombiliti is currently manufactured in the People's Republic of China by WuXi Biologics Co., Ltd. The company is working with WuXi to develop a second manufacturing facility for Pombiliti in Dundalk, Ireland. This facility is EMA approved, but there can be no assurance it will be approved by the FDA or other regulatory bodies. The company has implemented appropriate controls for assuring the quality of both active biopharmaceutical ingredients and final drug products.

The company's capital allocation priorities include funding research and development, commercialization activities, and capital expenditures. For the year ended December 31, 2025, research and development expense was $135.8 million ²¹, which included a $30.0 million ²² upfront license payment to Dimerix. Capital expenditures were $3.3 million ²³ for the year ended December 31, 2025. As of December 31, 2025, an aggregate of $164.2 million ²⁴ worth of shares remain available to be issued and sold under the at-the-market equity program, though the company is prohibited from offering additional shares under the Merger Agreement. The company does not anticipate paying cash dividends in the foreseeable future.

A significant headwind is the pending transaction with BioMarin, which may not be completed within the anticipated timeframe or at all. The Merger Agreement may be terminated under certain circumstances, and the company could be required to pay BioMarin a termination fee of $175 million ²⁵. The pendency of the transaction could cause disruptions, including diverting management attention and affecting employee retention. While the Merger Agreement is in effect, the company is subject to restrictions on business activities, including limitations on incurring additional indebtedness, issuing additional shares, and making capital expenditures above specified thresholds.

The company faces risks from foreign currency exchange rate fluctuations, as the majority of its Galafold and Pombiliti + Opfolda revenue is derived from outside the U.S. The company is not currently engaged in any foreign currency hedging activities. A near-term 10% fluctuation of the U.S. dollar to GBP exchange rate could result in a potential change in the fair value of net GBP denominated assets and liabilities of approximately $48.6 million ²⁶. A near-term 10% fluctuation of the U.S. dollar to EUR exchange rate could result in a potential change in the fair value of net EUR denominated assets and liabilities of approximately $28.5 million ²⁷.

The company depends heavily on sales of Galafold and Pombiliti + Opfolda, and if it is unable to commercialize these products successfully, its business could be materially harmed. The company faces substantial competition from major pharmaceutical companies with approved therapies for Fabry disease (Sanofi, Takeda, Chiesi) and Pompe disease (Sanofi), as well as from potential generic versions of Galafold. The company has entered into license agreements with Teva, Aurobindo, and Lupin allowing them to market generic versions of Galafold in the U.S. beginning on January 30, 2037 ²⁸, or earlier in certain circumstances. The company relies on third-party manufacturers, including WuXi Biologics in the People's Republic of China as the primary supplier of Pombiliti, which exposes it to risks related to geopolitical tensions, trade disputes, and potential sanctions. The company has incurred significant losses since inception, with an accumulated deficit of $2.8 billion ²⁹ as of December 31, 2025, and may never become profitable. The company has substantial debt of $400 million ³⁰ under the Senior Secured Term Loan due 2029, which requires interest-only payments until early-2027 and contains a minimum liquidity covenant and a minimum consolidated revenue covenant. The pending transaction with BioMarin may not be completed, and the company could be required to pay a termination fee of $175 million ³¹ under certain circumstances.

Management's message emphasizes the company's mission to develop and deliver transformative medicines for people living with rare diseases, with a strategy to create, manufacture, test, and deliver the highest quality medicines through internally developed, jointly developed, acquired, or in-licensed products. Key themes include commercial success in Fabry disease with Galafold revenue of $521.7 million ³² for the year ended December 31, 2025, representing an increase of $63.6 million compared to the prior year; commercial and regulatory success in Pompe disease with Pombiliti + Opfolda revenue of $112.5 million ³³ for the year ended December 31, 2025, representing an increase of $42.3 million compared to the prior year; pipeline advancement through the licensing of DMX-200; and financial strength with total cash, cash equivalents, and marketable securities of $293.5 million ³⁴ as of December 31, 2025. The strategic priorities emphasized for the period ahead include continuing to launch Galafold in additional countries, pursuing regulatory submissions and reimbursement processes for Pombiliti + Opfolda globally, and preparing for the U.S. commercial launch of DMX-200, should it be approved by the FDA.