• Amicus is like BioMarin Pharmaceutical, but specializing in treatments for rare lysosomal storage disorders.
• Amicus is like Sarepta Therapeutics, but focusing on a range of rare lysosomal storage diseases with gene therapies and other advanced treatments.

• Galafold (migalastat): An oral precision medicine for the treatment of Fabry disease in adults and adolescents aged 12 years and older with a confirmed amenable GLA mutation.
• AT-GAA (cipaglucosidase alfa and miglustat): A two-component therapy, consisting of an enzyme replacement therapy and an enzyme stabilizer, for the treatment of Pompe disease.

Amicus Therapeutics (FOLD) - Major Customers

Amicus Therapeutics is a biotechnology company focused on the development and commercialization of medicines for people living with rare diseases. As is typical for pharmaceutical companies, Amicus Therapeutics primarily sells its products to other companies, which then distribute and dispense the treatments to patients, rather than selling directly to individuals.

Their commercialization strategy involves selling to a limited number of specialty distributors, specialty pharmacies, hospitals, and clinics across various global markets.

According to Amicus Therapeutics' annual report (10-K filing for the year ended December 31, 2023), the company does have significant customer concentration, indicating "major customers" in terms of revenue. Specifically, their filing states:

• Three customers individually accounted for 42% of Amicus Therapeutics' consolidated gross product sales for the year ended December 31, 2023.
• These three customers also individually accounted for 64% of their consolidated accounts receivable as of December 31, 2023.

However, Amicus Therapeutics does not disclose the specific names of these major customers in their public filings. This practice is common in the pharmaceutical industry due to competitive and proprietary reasons.

Based on Amicus Therapeutics' described sales channels and typical industry distribution models for specialized rare disease treatments, these undisclosed major customers are highly likely to be large pharmaceutical wholesalers, specialty distributors, significant specialty pharmacy chains, or major hospital systems that procure and distribute their products. While not confirmed as Amicus Therapeutics' specific major customers, prominent pharmaceutical wholesalers in the U.S. market often include:

• AmerisourceBergen (NYSE: ABC)
• Cardinal Health (NYSE: CAH)
• McKesson Corporation (NYSE: MCK)

It is important to reiterate that Amicus Therapeutics has not publicly identified the specific names of its major customers.

• Thermo Fisher Scientific (TMO)

Bradley L. Campbell President and Chief Executive Officer

Bradley L. Campbell joined Amicus Therapeutics in 2006, serving as President & Chief Operating Officer before being appointed President and Chief Executive Officer. Prior to Amicus, he held various commercial and business development roles at Genzyme and Bristol-Myers Squibb. He began his career as an analyst at Marakon, a global management consulting firm. Mr. Campbell also served on the board of Progenics Pharmaceuticals from 2016 until its acquisition by Lantheus Holdings in 2020.

Simon Harford Chief Financial Officer

Simon Harford joined Amicus Therapeutics as Chief Financial Officer in August 2023. His previous experience includes serving as Chief Financial Officer of Boston-based biotech Albireo Pharma Inc. and Chief Financial Officer at PAREXEL International Corporation. He also held leadership positions at GlaxoSmithKline plc and Eli Lilly and Company.

John F. Crowley Chairman Emeritus

John F. Crowley is the Founder and Executive Chairman Emeritus of Amicus Therapeutics, having served as its CEO from 2005 to 2022. His involvement in biotechnology began after two of his children were diagnosed with Pompe disease. In his quest for a cure, he co-founded Novazyme Pharmaceuticals, a biotech startup. Novazyme was acquired by Genzyme Corporation in 2001, where Mr. Crowley continued to lead drug development for Pompe disease as Senior Vice President. He also founded Orexigen Therapeutics in 2003.

Jeffrey P. Castelli Chief Development Officer

Jeffrey P. Castelli joined Amicus Therapeutics in July 2005 and currently serves as Chief Development Officer, and also holds the title of Chief Portfolio Officer and Head of Gene Therapy. He has over 20 years of experience in the biotech and orphan drug industry, focusing on rare disease research and development. Prior to Amicus, he worked as a healthcare strategy consultant at Health Advances LLC and in business development at Neose Pharmaceuticals Inc.

Hung Do Chief Science Officer

Hung Do, Ph.D., joined Amicus Therapeutics in December 2013 and became Chief Science Officer in July 2015. He previously co-founded and served as Chief Scientific Officer of Callidus Biopharma, a privately-held biologics company, which was acquired by Amicus Therapeutics in November 2013. Before founding Callidus, Dr. Do headed early discovery research at Amicus and worked at Genzyme and Novazyme, focusing on lysosomal diseases and enzyme replacement therapies.

Key Risks to Amicus Therapeutics (FOLD)

1. Heavy Reliance on Key Therapies and Pipeline Risk: Amicus Therapeutics exhibits a substantial reliance on a limited number of high-value therapies, notably Galafold. This concentration in its product portfolio and the absence of a deep pipeline present a considerable risk. Should any of these primary drugs encounter issues such as new competition, unforeseen safety concerns, or shifts in market demand, the company's revenue and overall business stability could be significantly impacted.
2. Profitability Challenges and High Leverage: Despite maintaining robust gross margins, Amicus Therapeutics continues to grapple with profitability challenges, reflected in negative pretax and net profit margins. This indicates ongoing operational inefficiencies that impact its ability to convert revenue into net income. Furthermore, the company carries a high level of leverage, with a debt-to-equity ratio of 1.92 and substantial long-term debt of $434 million, signaling potential financial instability and concerns regarding its long-term financial health and ability to generate shareholder returns.
3. Competition: Amicus Therapeutics operates in a competitive market, facing headwinds from existing and emerging rivals. Specifically, the company contends with competition from larger pharmaceutical companies, such as Sanofi, which markets Fabrazyme for the treatment of Fabry disease, a key therapeutic area for Amicus. This competitive pressure can affect market share, pricing power, and the overall revenue potential of Amicus's products.

The primary clear emerging threat to Amicus Therapeutics stems from the advancement of gene therapies for Fabry disease, which is the indication for Amicus's lead commercial product, Galafold (migalastat).

Galafold is an oral chaperone therapy that requires chronic administration. In contrast, gene therapies aim to provide a single, potentially curative treatment by introducing a functional copy of the deficient gene. This represents a fundamental shift in the treatment paradigm from chronic disease management to a potential long-term genetic correction.

Several companies are actively developing gene therapies for Fabry disease, with candidates already in clinical trials. Examples include programs from 4D Molecular Therapeutics (e.g., 4D-310) and Sangamo Therapeutics (e.g., ST-920). If these gene therapy candidates demonstrate favorable efficacy and safety profiles in later-stage clinical development, they could significantly disrupt the market for existing chronic Fabry treatments, including Galafold, by offering a potentially superior, one-time treatment option.

Amicus Therapeutics (symbol: FOLD) focuses on developing and commercializing therapies for rare and orphan diseases, primarily lysosomal storage disorders. Their main commercialized products are Galafold (migalastat) for Fabry disease and Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) for Pompe disease. The addressable markets for these products are as follows:

Galafold (Fabry Disease)

The global addressable market for Fabry disease treatment is estimated to be approximately $2.63 billion USD in 2025 and is projected to reach approximately $3.87 billion USD by 2030. Another estimate places the global market at $2.4 billion USD in 2025, with a projection to reach $5.2 billion USD by 2035. This market size is for the global region. Amicus Therapeutics estimates that approximately 35% to 50% of individuals living with Fabry disease have amenable GLA variants, which Galafold targets. Amicus Therapeutics held approximately 69% of the global market share of treated Fabry patients with amenable mutations at the end of Q3 2025.

Pombiliti + Opfolda (Pompe Disease)

The global addressable market for Pompe disease treatment is estimated to be approximately $1.91 billion USD in 2025 and is forecast to expand to approximately $2.72 billion USD by 2030. This market size is for the global region. Another report indicates the global market was valued at $3.7 billion USD in 2024 and is expected to reach approximately $7.96 billion USD by 2035.

Amicus Therapeutics anticipates surpassing $1 billion in total sales by 2028 from its current commercial business with Galafold and Pombiliti + Opfolda.

1. Continued Growth of Commercial Products (Galafold and Pombiliti + Opfolda): Amicus anticipates sustained strong demand and increased patient starts for its two primary commercial therapies. Galafold, for Fabry disease, is projected to achieve 10-15% revenue growth in 2025. Pombiliti + Opfolda, for Pompe disease, is expected to see even more significant growth, with projections of 50-65% revenue growth in 2025. This growth is driven by continued commercial execution in all markets, new patient starts, and strong compliance. The company aims to increase Galafold's share of treated amenable patients, aspiring to reach 85-90% in most mature markets globally.
2. Global Market Expansion and New Reimbursement Agreements: Amicus is actively expanding its global presence by securing new pricing and reimbursement agreements in additional countries. Recent agreements have been established in countries like Italy, Sweden, Switzerland, and the Czech Republic, with first commercial patients anticipated in the first half of 2025. The company also expects new regulatory decisions in Australia, Canada, and Japan in 2025, further broadening its international revenue base. This geographic expansion is seen as a key strategy to increase patient reach and drive sustained long-term revenue growth.
3. Increased Patient Identification and Penetration: A significant driver involves finding new patients and improving diagnostic pathways for both Fabry and Pompe diseases. Amicus highlights a larger underdiagnosed and untreated patient population than previously estimated, offering substantial room for growth through increased patient penetration and shortening the pathway to diagnosis. The company's focus is on expanding patient populations for its existing therapies.
4. Advancement of Pipeline Candidates (e.g., DMX-200): Amicus is advancing its late-stage pipeline, which includes DMX-200 for Focal Segmental Glomerulosclerosis (FSGS). Progress in these pipeline assets, with clear regulatory alignment and clinical differentiation in areas of high unmet medical need, is expected to increase the company's prospects for future earnings growth and product diversification beyond its current commercial offerings.

Share Repurchases

• Amicus Therapeutics conducted approximately $58.0 million in share repurchases between June 2020 and March 2025.

Share Issuance

• In September 2021, Amicus Therapeutics received approximately $200 million through a private investment round from a syndicate of healthcare biotechnology investors as part of the Caritas Therapeutics formation.
• A Post IPO funding round in October 2023 raised $30 million from investors.

Inbound Investments

• Amicus Therapeutics received a private investment of approximately $200 million in September 2021 from a syndicate of healthcare biotechnology investors during the formation of Caritas Therapeutics.
• The company raised $30 million in a Post IPO funding round in October 2023.

Outbound Investments

• Amicus Therapeutics made a $30 million upfront payment to Dimerix for the U.S. commercialization rights to DMX-200.
• The company has licensing deals with the University of Pennsylvania, with potential aggregate outlays of $163 million, and a deal with GSK involving mid-teen royalties in eight territories outside the U.S.

Capital Expenditures

• Amicus Therapeutics' capital expenditures have historically focused on developing its gene therapy pipeline, expanding manufacturing capabilities for its Pompe biologic AT-GAA, and establishing the Amicus Process Science and Gene Therapy Manufacturing facility.