Amicus Therapeutics, Inc. (FOLD): History, Ownership, Mission, How It Works & Makes Money

Ever wondered how a biotech company dedicated to rare diseases operates and generates revenue? Amicus Therapeutics, Inc. (FOLD), a global, patient-dedicated biotechnology company, focuses on discovering, developing, and delivering novel high-quality medicines for people living with rare diseases. Despite a 30% stock decline since 2021, is Amicus Therapeutics poised for a comeback, driven by its revenue growth and market potential?

With $528.30 million in revenue for 2024, a 32.29% increase from the previous year, and losses significantly reduced by -62.99% to -$56.11 million, let's delve into the history, ownership, mission, and operational strategies of Amicus Therapeutics to understand its financial workings and future prospects.

Amicus Therapeutics, Inc. (FOLD) History

[Company's] Founding Timeline

Year established

The company was founded in 2002.

Original location

The company was originally located in Cranbury, New Jersey.

Founding team members

The founding team included:

• John F. Crowley

Initial capital/funding

In 2002, the company secured $10.5 million in Series A financing led by Radius Ventures.

[Company's] Evolution Milestones

[Company's] Transformative Moments

• Early Focus on Chaperone Technology: The company's initial focus on developing pharmacological chaperones to treat lysosomal storage disorders set the stage for its innovative approach.
• Pivotal Clinical Trials: Positive results from clinical trials of Galafold were crucial in securing regulatory approvals and establishing the drug as a viable treatment option.
• Strategic Acquisitions: Acquisitions like Callidus BioSciences broadened the company's portfolio and capabilities in gene therapy, positioning it for future growth.
• Navigating Regulatory Hurdles: Successfully navigating the regulatory landscape in both Europe and the United States was a transformative moment, leading to the commercialization of Galafold.

To gain more insights into the values that drive the company, explore: Mission Statement, Vision, & Core Values of Amicus Therapeutics, Inc. (FOLD).

Amicus Therapeutics, Inc. (FOLD) Ownership Structure

Amicus Therapeutics, Inc. has a mixed ownership structure, comprising institutional, retail, and insider stakeholders. The distribution of shares among these groups influences the company's governance and strategic direction.

Amicus Therapeutics, Inc.'s Current Status

Amicus Therapeutics, Inc. is a public company, trading on the NASDAQ Global Select Market under the ticker symbol FOLD. As a publicly traded entity, it is subject to regulations and reporting requirements set forth by the U.S. Securities and Exchange Commission (SEC).

Amicus Therapeutics, Inc.'s Ownership Breakdown

Understanding the ownership structure of Amicus Therapeutics provides insights into who has influence over the company’s decisions. Here’s a breakdown:

For more detailed insights into Amicus Therapeutics' financial standing, see Breaking Down Amicus Therapeutics, Inc. (FOLD) Financial Health: Key Insights for Investors.

Amicus Therapeutics, Inc.'s Leadership

The leadership team at Amicus Therapeutics is responsible for the strategic direction and operational management of the company. Key members of the leadership team include:

• John F. Crowley: Chairman and Chief Executive Officer
• Bradley L. Campbell: President and Chief Operating Officer
• Jeff Castelli: Chief Medical Officer
• Frederick J. Fry: Chief Global Supply Chain Officer
• Ellen K. Rosenberg: General Counsel

These individuals bring a wealth of experience in the biotechnology and pharmaceutical industries, guiding Amicus Therapeutics in its mission to develop and deliver treatments for rare diseases.

Amicus Therapeutics, Inc. (FOLD) Mission and Values

Amicus Therapeutics is dedicated to developing and delivering therapies for rare diseases, focusing on improving the lives of individuals and families affected by these conditions. The company's values emphasize innovation, collaboration, and a patient-centric approach.

Amicus Therapeutics' Core Purpose

Official mission statement

Amicus Therapeutics' mission statement is to discover, develop, and deliver innovative medicines for people living with rare diseases. This mission drives the company's research and development efforts, as well as its commitment to patient advocacy and support. More information about the company's mission and vision can be found at: Mission Statement, Vision, & Core Values of Amicus Therapeutics, Inc. (FOLD).

• Focus on rare diseases: Targeting conditions with limited or no treatment options.
• Innovative medicines: Developing novel therapies to address the underlying causes of rare diseases.
• Patient-centric approach: Prioritizing the needs and well-being of patients and their families.

Vision statement

Amicus Therapeutics envisions a world where no one living with a rare disease is beyond reach of a therapy. This vision reflects the company's ambition to expand its portfolio of treatments and make a meaningful difference in the lives of rare disease patients globally.

• Global impact: Expanding access to therapies for rare disease patients worldwide.
• Broad portfolio: Developing treatments for a wide range of rare diseases.
• Transformative therapies: Creating medicines that can significantly improve patient outcomes.

Company slogan/tagline

While specific slogans or taglines may vary over time, Amicus Therapeutics consistently communicates its commitment to rare disease patients through its actions and messaging. The company's focus on innovation, patient advocacy, and collaboration underscores its dedication to making a positive impact on the rare disease community.

• Innovation: Continuously seeking new and better ways to treat rare diseases.
• Patient Advocacy: Supporting and empowering patients and their families.
• Collaboration: Working with researchers, healthcare providers, and patient organizations to advance rare disease research and treatment.

Amicus Therapeutics, Inc. (FOLD) How It Works

Amicus Therapeutics, Inc. is a biotechnology company focused on discovering, developing, and delivering medicines for rare diseases.

Amicus Therapeutics, Inc.'s Product/Service Portfolio

Amicus Therapeutics, Inc.'s Operational Framework

Amicus Therapeutics operates through an integrated framework encompassing research and development, clinical trials, regulatory approvals, manufacturing, and commercialization. Key aspects of its operational framework include:

• Research and Development: Amicus focuses on identifying and developing novel therapies for rare diseases. This involves preclinical research, drug discovery, and formulation development.
• Clinical Trials: Rigorous clinical trials are conducted to evaluate the safety and efficacy of drug candidates. These trials adhere to strict regulatory guidelines and involve multiple phases to ensure comprehensive data collection.
• Regulatory Approvals: Amicus works closely with regulatory agencies such as the FDA in the United States and the EMA in Europe to secure approvals for its therapies. This includes submitting detailed data packages and complying with all regulatory requirements.
• Manufacturing: The company manages the manufacturing process to ensure a consistent and high-quality supply of its products. This may involve internal manufacturing capabilities as well as partnerships with contract manufacturing organizations (CMOs).
• Commercialization: Amicus commercializes its approved therapies through a specialized sales and marketing team. This includes educating healthcare professionals, raising awareness among patients, and ensuring access to treatment.

Amicus Therapeutics, Inc.'s Strategic Advantages

Amicus Therapeutics has several strategic advantages that enable its success in the rare disease market:

• Focus on Rare Diseases: Amicus's exclusive focus on rare diseases allows it to develop deep expertise and understanding of specific disease areas, patient needs, and treatment landscapes.
• Innovative Therapies: The company's emphasis on developing innovative therapies, such as precision medicines and enzyme replacement therapies, provides a competitive edge and addresses unmet medical needs.
• Strong Intellectual Property: Amicus maintains a robust intellectual property portfolio to protect its drug candidates and approved therapies, ensuring market exclusivity and long-term revenue potential.
• Global Reach: With operations and partnerships around the world, Amicus has a global reach that allows it to commercialize its products in key markets and serve patients worldwide.
• Patient Advocacy: Amicus actively engages with patient advocacy groups to raise awareness, support research, and ensure that patient perspectives are integrated into its drug development and commercialization efforts.

To gain more insights into the company's values, read Mission Statement, Vision, & Core Values of Amicus Therapeutics, Inc. (FOLD).

Amicus Therapeutics, Inc. (FOLD) How It Makes Money

Amicus Therapeutics, Inc. primarily generates revenue through the sale of its commercial therapies, particularly Galafold, and to a lesser extent through collaborations and partnerships aimed at developing and commercializing novel treatments for rare diseases.

Amicus Therapeutics, Inc.'s Revenue Breakdown

Amicus Therapeutics, Inc.'s Business Economics

Amicus Therapeutics, Inc.'s business economics are driven by several key factors:

• Pricing Strategy: Amicus focuses on developing treatments for rare diseases, which often allows for premium pricing due to the lack of alternative therapies and the high unmet medical need.
• Market Access: Securing regulatory approvals and market access in major markets, including the United States and Europe, is crucial for maximizing revenue potential.
• Cost of Goods Sold (COGS): Managing the costs associated with manufacturing and distributing Galafold is essential for maintaining profitability.
• Research and Development (R&D) Expenses: A significant portion of Amicus's expenses is allocated to R&D, as the company invests in developing new therapies and expanding the applications of existing ones.
• SG&A Expenses: Selling, general, and administrative expenses, including marketing and sales costs, also impact the company's overall profitability.

Amicus Therapeutics, Inc.'s Financial Performance

Amicus Therapeutics, Inc.'s financial performance can be assessed through the following points:

• Revenue Growth: In 2024, Amicus Therapeutics reported a 22% increase in total revenue, reaching $526.4 million, driven primarily by the strong performance of Galafold.
• Gross Margin: The company's gross margin for 2024 was approximately 70%, reflecting efficient cost management in the production and distribution of Galafold.
• Operating Expenses: Amicus reported operating expenses of $483.7 million for 2024, which included $353.8 million in research and development expenses and $129.9 million in selling, general, and administrative expenses.
• Net Loss: Despite revenue growth, Amicus experienced a net loss of $151.7 million, or $0.53 per share, in 2024, due to substantial investments in research and development.
• Cash Position: As of December 31, 2024, Amicus had cash, cash equivalents, and marketable securities totaling $427.9 million, providing a solid foundation for future growth and development initiatives.

To gain more insights into investor behavior and ownership dynamics, check out: Exploring Amicus Therapeutics, Inc. (FOLD) Investor Profile: Who’s Buying and Why?

Amicus Therapeutics, Inc. (FOLD) Market Position & Future Outlook

As of April 2025, Amicus Therapeutics is navigating a competitive biopharmaceutical landscape, focusing on rare diseases with its innovative therapies and gene therapy advancements; details about its market capitalization and enterprise value can offer insights into its financial health and investor confidence. Exploring Amicus Therapeutics, Inc. (FOLD) Investor Profile: Who’s Buying and Why?

Competitive Landscape

Opportunities & Challenges

Industry Position

Amicus Therapeutics holds a significant position within the biopharmaceutical industry, particularly in the realm of rare genetic diseases. This standing is supported by several factors:

• Niche Focus: The company's concentration on developing therapies for rare diseases allows it to address unmet medical needs, often with limited competition.
• Innovative Pipeline: Amicus is actively involved in advancing gene therapy technologies, positioning it at the forefront of potential breakthroughs in treating genetic disorders.
• Global Reach: Amicus has expanded its market presence internationally, ensuring that its therapies are accessible to patients worldwide.