Amicus Therapeutics, Inc. (Fold): Modelo de negócios Canvas [Jan-2025 Atualizado]

No mundo de ponta da terapêutica de doenças raras, a Amicus Therapeutics (dobra) surge como um farol de esperança, transformando distúrbios genéticos complexos por meio de medicina inovadora de precisão. Ao alavancar a biotecnologia avançada e as parcerias estratégicas, esta empresa pioneira está redefinindo as possibilidades de tratamento para pacientes com opções médicas limitadas, oferecendo terapias inovadoras que visam mutações genéticas específicas e potencialmente revolucionar o atendimento ao paciente. Seu modelo de modelo de negócios exclusivo revela uma abordagem sofisticada para o desenvolvimento de medicamentos, combinando a excelência científica com uma missão profundamente centrada no paciente que promete desbloquear novas fronteiras em tratamentos médicos personalizados.

Amicus Therapeutics, Inc. (dobra) - Modelo de negócios: Parcerias -chave

Colaborações estratégicas com empresas farmacêuticas

A Amicus Therapeutics estabeleceu parcerias estratégicas com as principais empresas farmacêuticas:

Empresa parceira	Detalhes da parceria	Ano estabelecido
GlaxoSmithKline (GSK)	Colaboração de terapia de doenças de Fabry	2010
JCR Pharmaceuticals	Parceria de Desenvolvimento Global para Migalastat	2014

Parcerias de pesquisa com instituições acadêmicas

Amicus Therapeutics colabora com as principais instituições de pesquisa:

• Universidade da Pensilvânia - Pesquisa de Doenças Raras
• Escola de Medicina de Harvard - Estudos de dobramento de proteínas
• Hospital Geral de Massachusetts - Programas de Pesquisa Clínica

Organizações de fabricação contratadas

Parceiro da CMO	Foco de fabricação	Valor do contrato
Grupo Lonza	Fabricação em escala comercial para Galafold	Contrato anual de US $ 35 milhões
Patheon	Produção de medicamentos em estágio clínico	Contrato de fabricação de US $ 22 milhões

Acordos de licenciamento para terapias de doenças raras

As principais parcerias de licenciamento incluem:

• Licença exclusiva mundial para Migalastat da GlaxoSmithKline
• Contrato de licenciamento de terapia de reposição enzimática com a JCR Pharmaceuticals

Parcerias de rede de ensaios clínicos globais

Amicus Therapeutics mantém parcerias de ensaios clínicos:

• América do Norte: 27 sites de pesquisa
• Europa: 18 sites de pesquisa
• Ásia-Pacífico: 12 sites de pesquisa

Rede de parceria total: 57 locais de pesquisa e ensaios clínicos globais

Amicus Therapeutics, Inc. (dobra) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de drogas de doenças raras

A partir do quarto trimestre de 2023, a Amicus Therapeutics investiu US $ 305,7 milhões em despesas de pesquisa e desenvolvimento. A empresa se concentra no desenvolvimento de terapias para doenças genéticas raras, com uma ênfase específica na doença de Fabry e na doença de Pompe.

Área de foco de pesquisa	Investimento (2023)	Programas de pesquisa ativa
Distúrbios genéticos raros	US $ 305,7 milhões	4 programas terapêuticos primários

Desenvolvimento de Medicina de Precisão para Distúrbios Genéticos

Amicus Therapeutics desenvolveu 3 plataformas de medicina de precisão direcionando mutações genéticas específicas.

• Tecnologia de reposição com avanços de acompanhante (gráfico)
• Otimização da terapia de reposição enzimática (ERT)
• Abordagens terapêuticas específicas da mutação genética

Gerenciamento e execução de ensaios clínicos

Em 2023, a empresa administrou 7 ensaios clínicos ativos em vários distúrbios genéticos raros.

Fase de ensaios clínicos	Número de ensaios	Inscrição do paciente
Fase I.	2 ensaios	87 pacientes
Fase II	3 ensaios	215 pacientes
Fase III	2 ensaios	342 pacientes

Pesquisa farmacológica em terapias de reposição enzimática

A Amicus Therapeutics alocou US $ 178,4 milhões especificamente para pesquisas de terapia de reposição enzimática em 2023.

• Pesquisa Migalastat para Doença Fabry
• Formulações avançadas de ERT
• Desenvolvimento da tecnologia de chaperona molecular

Processos de conformidade regulatória e aprovação de medicamentos

A Companhia manteve a conformidade com os padrões regulatórios da FDA e da EMA, com 2 novas solicitações de drogas enviadas em 2023.

Agência regulatória	Envios	Aprovações
FDA	1 nova aplicação de drogas	1 designação de medicamentos órfãos
Ema	1 nova aplicação de drogas	1 designação de medicamentos órfãos

Amicus Therapeutics, Inc. (dobra) - Modelo de negócios: Recursos -chave

Capacidades avançadas de pesquisa de biotecnologia

A partir do quarto trimestre de 2023, a Amicus Therapeutics investiu US $ 274,1 milhões em despesas de pesquisa e desenvolvimento. A empresa mantém instalações de pesquisa avançadas focadas em doenças genéticas raras.

Investimento em pesquisa	2023 quantidade
Despesas de P&D	US $ 274,1 milhões
Instalações de pesquisa	3 centros de pesquisa primários

Portfólio de propriedade intelectual

A Amicus Therapeutics se aplica 42 patentes ativas Relacionado a tratamentos de doenças raras em dezembro de 2023.

• Patentes raros de tratamento de doenças genéticas: 42
• Aplicações de patentes pendentes: 18
• Áreas de foco terapêutico primário: doença de Fabry, doença de Pompe

Talento científico e médico especializado

Categoria de força de trabalho	Número
Total de funcionários	595
Pesquisadores no nível de doutorado	127
Especialistas em pesquisa clínica	86

Plataformas de tecnologia proprietárias

A plataforma de terapia de reposição avançada com acompanhante (gráfico) representa um Principais ativos tecnológicos Para desenvolver terapias de reposição enzimática.

Recursos financeiros

Métrica financeira	2023 valor
Caixa e equivalentes de dinheiro	US $ 692,3 milhões
Total de ativos	US $ 1,2 bilhão
Receita anual	US $ 330,5 milhões

Amicus Therapeutics, Inc. (dobra) - Modelo de negócios: proposições de valor

Terapias inovadoras para distúrbios genéticos raros

Amicus Therapeutics se concentra no desenvolvimento de terapias para distúrbios genéticos raros com características específicas do mercado:

Categoria de doença rara	Número de condições direcionadas	População estimada de pacientes
Distúrbios de armazenamento lisossômicos	3 condições primárias	Aproximadamente 10.000 a 15.000 pacientes globalmente
Doença de Fabry	1 foco primário	Estimado 5.000 a 10.000 pacientes em todo o mundo

Abordagens de medicina personalizadas

Amicus Therapeutics implementa estratégias de medicina de precisão com as seguintes capacidades tecnológicas:

• Plataforma de tecnologia de chaperona farmacológica
• Mecanismos de direcionamento de mutação genética avançada
• Otimização de tratamento específica do paciente

Tratamentos potenciais que mudam a vida

O investimento financeiro em pesquisa e desenvolvimento demonstra compromisso com os tratamentos inovadores:

Investimento em pesquisa	2023 Despesas de P&D	Investimentos em estágio de ensaios clínicos
Gastos totais de P&D	US $ 354,2 milhões	Fase 2-3 Ensaios Clínicos: US $ 198,6 milhões

Tecnologias terapêuticas avançadas

Principais plataformas tecnológicas que impulsionam a proposta de valor:

• Terapia de reposição da enzima AT-GAA para doença de Pompe
• Migalastat Precision Medicine abordagem
• Tecnologias avançadas de segmentação de mutação genética

Melhor qualidade de vida

Métricas de impacto do paciente para distúrbios genéticos raros direcionados:

Doença	Eficácia do tratamento	Benefício potencial do paciente
Doença de Fabry	Melhoria da atividade enzimática: 50-75%	Progressão reduzida de danos nos órgãos
Doença de Pompe	Melhoria da função do motor: 30-45%	Mobilidade aprimorada e expectativa de vida

Amicus Therapeutics, Inc. (dobra) - Modelo de negócios: relacionamentos com o cliente

Engajamento direto com comunidades de pacientes

A Amicus Therapeutics mantém o envolvimento direto do paciente por meio de programas direcionados para doenças genéticas raras. Em 2023, a empresa informou:

Programa da comunidade de pacientes	Métricas de engajamento
Rede de Suporte a Doenças Fabry	3.247 participantes ativos dos pacientes
Plataforma da comunidade de doenças de Pompe	2.189 membros do paciente registrado

Educação e apoio profissional médico

A empresa fornece recursos abrangentes de educação médica:

• 37 Simpósios médicos realizados em 2023
• 642 profissionais de saúde treinados em terapias genéticas raras
• Desenvolveu 18 módulos especializados de treinamento clínico

Programas de assistência ao paciente

Os programas de apoio financeiro incluem:

Programa de Assistência	Detalhes da cobertura
Migalastat Copay Support	Até US $ 20.000 para assistência anual do paciente
Suporte de terapia de reposição enzimática	Ajuda financeira anual máxima de US $ 15.000

Comunicação clínica em andamento

Canais de comunicação com pacientes e profissionais:

• 4 plataformas dedicadas de comunicação clínica
• Série semanal de webinar com 1.287 participantes médios
• Atualizações de ensaios clínicos em tempo real via portais digitais

Atualizações transparentes de pesquisa e desenvolvimento

Métricas de comunicação de pesquisa para 2023:

Canal de comunicação	Estatísticas de engajamento
Apresentações de pesquisa pública	12 Conferências Internacionais
Newsletter de pesquisa digital	8.456 assinantes
Portal de transparência de ensaio clínico	Classificação de acessibilidade de informações de 94%

Amicus Therapeutics, Inc. (Fold) - Modelo de Negócios: Canais

Força de vendas direta para medicamentos para doenças raras

A Amicus Therapeutics mantém uma equipe de vendas direta especializada de 85 representantes de vendas focados em doenças genéticas raras no quarto trimestre 2023.

Métrica da equipe de vendas	2024 dados
Total de representantes de vendas	85
Cobertura geográfica	Estados Unidos
Tamanho médio do território de vendas	3-4 estados por representante

Conferências médicas especializadas

A Amicus Therapeutics participa de 12-15 conferências médicas de doenças raras anualmente.

• Sociedade Americana de Gene & Conferência de terapia celular
• Organização Nacional para Distúrbios Raros (Nord) Cúpula
• Conferência Internacional de Doenças Lisossômicas

Portais de pacientes e médicos online

Métricas de engajamento da plataforma digital para 2024:

Métrica portal	Quantidade
Médicos registrados	2,300
Usuários mensais de pacientes ativos	1,750
Interações digitais anuais	42,000

Redes de distribuição farmacêutica

Parceiros de distribuição -chave:

• Amerisourcebergen
• Cardinal Health
• McKesson Corporation

Plataformas de comunicação em saúde digital

Estatísticas da plataforma de comunicação digital para 2024:

Métrica da plataforma	Quantidade
Seguidores de mídia social	37,500
Assinantes de newsletter por e -mail	8,200
Participantes do webinar anualmente	1,600

Amicus Therapeutics, Inc. (dobra) - Modelo de negócios: segmentos de clientes

Pacientes com distúrbios genéticos raros

Amicus Therapeutics tem como alvo pacientes com distúrbios genéticos raros específicos, incluindo:

• Doença de Fabry: aproximadamente 10.000 pacientes diagnosticados globalmente
• Doença de Pompe: estimado 5.000 a 10.000 pacientes em todo o mundo
• Distúrbios genéticos raros que afetam aproximadamente 30 milhões de pacientes nos Estados Unidos

Transtorno	População de pacientes	Prevalência global
Doença de Fabry	10.000 pacientes diagnosticados	1 em 40.000-60.000 nascimentos
Doença de Pompe	5.000 a 10.000 pacientes	1 em 40.000 nascidos vivos

Médicos especializados

Especialistas médicos -alvo incluem:

• Especialistas genéticos: 4.500 geneticistas clínicos nos Estados Unidos
• Especialistas em doenças metabólicas: aproximadamente 2.000 especialistas globalmente
• Médicos de doenças raras: estimado 8.000 profissionais em todo o mundo

Instituições de pesquisa de transtornos genéticos

Tipo de instituição	Número de instituições	Financiamento anual de pesquisa
Centros de pesquisa acadêmica	350 centros especializados	US $ 1,2 bilhão em pesquisa de doenças raras
Fundamentos de pesquisa de doenças raras	250 organizações dedicadas	Investimento anual de US $ 750 milhões

Sistemas de saúde com foco em doenças raras

Principais segmentos do sistema de saúde:

• Centros especializados de tratamento de doenças raras: 120 globalmente
• Clínicas abrangentes de transtorno genético: 85 na América do Norte
• Redes de tratamento de doenças raras: 45 redes internacionais

Organizações globais de apoio aos pacientes

Tipo de organização	Número de organizações	Alcance do paciente
Grupos de apoio a doenças raras	500+ organizações globais	Mais de 1,2 milhão de pacientes registrados
Redes de defesa de transtorno genético	250 redes internacionais	750.000 membros ativos

Amicus Therapeutics, Inc. (dobra) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, a Amicus Therapeutics registrou despesas de P&D de US $ 342,1 milhões. O foco de pesquisa da empresa tem como alvo principalmente doenças genéticas raras.

Ano	Despesas de P&D	% da receita total
2022	US $ 328,5 milhões	68.3%
2023	US $ 342,1 milhões	71.2%

Investimentos de ensaios clínicos

Os investimentos em ensaios clínicos para a Amicus Therapeutics em 2023 totalizaram aproximadamente US $ 187,6 milhões, cobrindo vários programas terapêuticos de doenças raras.

• Ensaios clínicos de doença de Fabry: US $ 62,4 milhões
• Ensaios clínicos da doença de Pompe: US $ 55,2 milhões
• Outros distúrbios genéticos raros: US $ 70 milhões

Custos de conformidade regulatória

As despesas de conformidade regulatória para 2023 foram estimadas em US $ 45,3 milhões, representando investimentos críticos na manutenção de aprovações regulatórias.

Despesas de fabricação e produção

Os custos de fabricação de 2023 atingiram US $ 89,7 milhões, com foco na produção biofarmacêutica especializada.

Categoria de produção	Despesa
Manutenção da instalação	US $ 24,5 milhões
Equipamento	US $ 35,2 milhões
Matérias-primas	US $ 30 milhões

Overhead administrativo e operacional

As despesas administrativas de 2023 foram de US $ 76,4 milhões, cobrindo operações corporativas e infraestrutura de gerenciamento.

• Custos de pessoal: US $ 52,1 milhões
• Infraestrutura de tecnologia: US $ 12,3 milhões
• Instalações corporativas: US $ 12 milhões

Amicus Therapeutics, Inc. (dobra) - Modelo de negócios: fluxos de receita

Vendas farmacêuticas de produtos

Para o ano fiscal de 2023, a Amicus Therapeutics registrou receita total de US $ 311,4 milhões. Principal Receita de Receita do Produto:

Produto	Receita anual
Galafold (tratamento de doenças de Fabry)	US $ 239,4 milhões
SD-101 (epidermólise bollosa)	US $ 72,0 milhões

Acordos de pesquisa colaborativa

A Amicus Therapeutics possui colaborações estratégicas de pesquisa gerando fluxos de receita adicionais.

• Parceria da GlaxoSmithKline gerando pagamentos marcantes
• Acordos de colaboração mundial em doenças genéticas raras

Propriedade intelectual de licenciamento

O licenciamento de propriedade intelectual gerou aproximadamente US $ 15,2 milhões em 2023.

Subsídios governamentais e de pesquisa

A receita da concessão de pesquisa para 2023 totalizou US $ 6,8 milhões de várias fontes de financiamento de pesquisa científica.

Potenciais pagamentos marcantes de parcerias

Parceria	Valor potencial de marco
GlaxoSmithKline Collaboration	Até US $ 1,2 bilhão
Outras parcerias farmacêuticas	Aproximadamente US $ 350 milhões

Amicus Therapeutics, Inc. (FOLD) - Canvas Business Model: Value Propositions

You're looking at the core reasons why patients and payers choose Amicus Therapeutics, Inc. (FOLD) products over alternatives in these niche, high-stakes rare disease markets. It's all about targeted efficacy and addressing true gaps in care. Here's the quick math on what they are delivering right now in late 2025.

Galafold: Oral, precision medicine for Fabry patients with amenable mutations

Galafold, the oral pharmacological chaperone for Fabry disease, continues to be the company's revenue workhorse. Its value proposition centers on being a precision medicine for adults with amenable galactosidase alpha gene (GLA) variants. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants. The drug's strong intellectual property provides patent protection in the U.S. until January 2037 following the settlement with Teva Pharmaceuticals. You can see the consistent financial traction:

Metric	Q3 2025 Value	Nine Months 2025 Value	Context/Growth
Net Product Sales	$138.3 million	$371.5 million	Reported growth of 15% year-over-year in Q3 2025.
Global Treated Patients	N/A	Approximately 2,730 individuals	Across more than 40 countries with regulatory approvals.
2025 Revenue Growth Guidance (CER)	10% to 15%		Reiterated guidance for the full year.

Pombiliti + Opfolda: Two-component therapy for late-onset Pompe disease

This two-component therapy is capturing significant momentum, especially in new launch countries. Its value is in providing an alternative or switch option for adults with late-onset Pompe disease. The commercial adoption is accelerating, as shown by the latest sales figures:

• Pombiliti + Opfolda Q3 2025 Net Product Sales were $30.7 million.
• Nine Months 2025 Net Product Sales reached $77.5 million.
• Q3 2025 saw a reported year-over-year increase of 45%, or 42% at Constant Exchange Rates (CER).
• As of the end of 2024, approximately 220 patients were treated or scheduled with the commercial product.
• The company updated 2025 revenue growth guidance to 50% to 65% at CER.

Addressing high unmet need in rare, life-threatening genetic diseases

Amicus Therapeutics, Inc. is focused on diseases where current options are insufficient or non-existent. This focus is translating into financial milestones, showing the market values these specialized solutions. The company achieved a major strategic objective by reporting GAAP profitability in the second half of 2025. Specifically, Q3 2025 delivered a GAAP Net Income of $17.3 million, reversing a loss from the prior year. The overall commercial strength supports a significant long-term financial target:

• Total Revenue Projection for 2028 is anticipated to surpass $1 billion.
• As of the end of 2024, over 3,000 people were treated with an Amicus therapy.

Potential first-in-class treatment (DMX-200) for FSGS, a rare kidney disease

The pipeline asset DMX-200 targets Focal Segmental Glomerulosclerosis (FSGS), a rare and fatal kidney disease for which the FDA has not yet approved a specific therapy. This represents a substantial unmet need. Amicus licensed the U.S. commercial rights, paying an upfront cash fee of $30 million to Dimerix. The clinical path has de-risking milestones:

• The pivotal Phase 3 ACTION3 study is on track for full enrollment by year-end 2025.
• In March 2025, the FDA aligned on proteinuria as an appropriate primary endpoint for traditional approval.
• Potential total consideration to Dimerix includes up to $560 million in success-based milestones.

Finance: finalize the 2025 revenue reconciliation against the updated guidance by Monday.

Amicus Therapeutics, Inc. (FOLD) - Canvas Business Model: Customer Relationships

You're focused on building deep, sustained connections in the rare disease space, which demands a very different approach than mass-market pharma. For Amicus Therapeutics, Inc., this means embedding support directly into the patient journey.

High-touch, specialized patient support programs (PSP) are central, exemplified by the Amicus Assist™ service, designed to provide product assistance and support to help patients gain access to Amicus Therapeutics' medications. This high-touch model supports the commercial base, which, as of Q2 2025, included approximately 2,730 individuals receiving Galafold across more than 40 countries.

Direct engagement with rare disease specialists and treatment centers is critical for driving adoption of therapies like Galafold and Pombiliti+Opfolda. Amicus Therapeutics reinforced its scientific leadership by showcasing 16 presentations (two oral presentations and 20 posters) at the 21st Annual WORLDSymposium™ in February 2025, focusing on Fabry and Pompe diseases. This scientific exchange supports the commercial base, with Galafold reaching 69% of treated amenable patients globally as of Q2 2025. Furthermore, the company is expanding access for Pombiliti+Opfolda, targeting more than 650 late-onset Pompe disease patients in new launch countries planned for 2025.

Long-term relationships with patient advocacy organizations are maintained through structured programs and charitable support. The Patient Advisory Board (PAB) Program was established in 2008 to enhance partnership and understanding of the patient community. Amicus Therapeutics provides charitable contributions to nonprofit organizations, including patient advocacy groups, focusing on educational, advocacy, and access initiatives related to the disease areas they focus on.

The structure supporting these relationships involves dedicated teams. Amicus Therapeutics' commercial staff, including field staff, are explicitly noted as not involved in decisions to fund Independent Medical Education (IME) programs, maintaining a clear boundary between commercial activity and medical education funding decisions. This structure helps ensure that interactions with healthcare professionals and community partners remain compliant with strict standards.

Here's a quick look at the scale of patient engagement and market reach as of mid-to-late 2025:

Metric	Value/Amount	Context/Date
Galafold Treated Patients (Global)	Approximately 2,730 individuals	Q2 2025
Galafold Global Market Penetration	69% of treated amenable patients	Q2 2025
Pombiliti+Opfolda New Launch Target Patients (2025)	More than 650 individuals	2025 Guidance
Scientific Presentations at WORLDSymposium	16 presentations	February 2025
Patient Advisory Board Program Established	2008	Long-term relationship foundation

The commitment is to build on this momentum, which is reflected in the Q3 2025 total revenue reaching $169 million, up 17% year-over-year at constant exchange rates.

You should review the current allocation of resources to the field teams to ensure they have the necessary support materials to facilitate these complex, high-touch patient interactions, especially with launches planned in up to 10 new countries in 2025. Finance: draft 13-week cash view by Friday.

Amicus Therapeutics, Inc. (FOLD) - Canvas Business Model: Channels

You're looking at how Amicus Therapeutics, Inc. (FOLD) gets its specialized, rare-disease medicines-Galafold and Pombiliti + Opfolda-into the hands of the patients who need them. For a company focused on niche markets, the channel strategy has to be precise; you can't just rely on broad distribution.

The core of the delivery mechanism relies on a highly specialized, global infrastructure, which is reflected in the revenue performance we're seeing through the third quarter of 2025. The scale of this operation is significant, supporting revenues that are growing double-digits year-over-year.

Direct Sales Force Targeting Specialized Physicians Globally

Amicus Therapeutics uses a direct sales force, which is essential when dealing with rare diseases where physician expertise is concentrated. This team targets the specialized centers and physicians who diagnose and manage Fabry and Pompe diseases. The success of this direct engagement is evident in the market penetration figures. For Galafold, which treats Fabry disease in patients with amenable genetic variants, Amicus Therapeutics has captured about 65% share of the treated amenable patient population globally. Furthermore, in the countries where the drug has been on the market the longest, they see the potential to reach 85% to 90% share, showing the effectiveness of their focused physician targeting and follow-up.

The growth of the newer therapy, Pombiliti + Opfolda, also relies on this specialized team, driving adoption across new launch countries. The company is committed to leveraging this 'unique and leverageable global rare disease organization' to support future pipeline assets, like the U.S. commercialization of DMX-200 for FSGS.

Specialty Pharmacies and Distributors for Drug Delivery

While the sales force drives prescription decisions, the physical delivery of these often-infusion or specialized oral therapies requires a tightly controlled network. This means relying on specialty pharmacies and distributors who can manage the complex logistics and patient support required for these treatments. The growth in patient numbers underscores the operational capacity of these channels. As of the end of 2024, approximately ~2,730 patients were on Galafold. By the third quarter of 2025, the company reported serving more patients than ever before, driven by new Galafold starts and growing adoption for Pombiliti + Opfolda. The scale of product movement is clear from the revenue figures:

Metric (As of Q3 2025)	Value	Context
Galafold Net Product Sales (Q3 2025)	$138.3 million	Reflects consistent demand and compliance in established markets.
Pombiliti + Opfolda Net Product Sales (Q3 2025)	$30.7 million	Represents a 45% year-over-year increase (42% at CER).
Nine Months 2025 Total Revenue	$448.998 million	Represents a 19% year-over-year growth reported.

Also, the supply chain itself is a channel consideration; Amicus Therapeutics is actively transitioning the Pombiliti drug product manufacturing supply from China to Ireland, with primary supply expected from Ireland by 2027.

Global Regulatory and Reimbursement Pathways (e.g., FDA, EMA, Japan)

Market access is the gatekeeper for these channels. Amicus Therapeutics has successfully navigated numerous global pathways. Galafold is currently approved in over 40 countries, including the U.S., EU, U.K., and Japan. For Pombiliti + Opfolda, the company secured regulatory approval in Canada and Australia for adult LOPD patients in Q1 2025, and granted approval in Japan in June 2025 for adult LOPD patients. The company continues to anticipate additional reimbursement agreements throughout the year, which directly impacts channel uptake. They remain on track for up to 10 new launch countries in 2025, which include markets with over 650 individuals living with LOPD.

The U.S. FDA approval for Pombiliti + Opfolda is anticipated early next year (2026), which will open up a major segment of the U.S. market for that therapy.

Digital and In-Person Medical Education for Healthcare Providers

To drive the 'net new patient starts' and the 'switch opportunity' for Galafold, ongoing medical education is key. This involves both digital dissemination of real-world evidence and in-person engagements with specialists. The growing body of real-world evidence for Pombiliti + Opfolda is a major educational focus point to accelerate adoption. For example, new analysis of the PROPEL study data was published in Muscle and Nerve, providing material for these educational efforts. The company is also focused on advancing ongoing studies to broaden labels and maintain scientific leadership in Fabry and Pompe diseases, which fuels the content for these educational channels.

The company is focused on delivering 2025 total revenue growth of 17% to 24% at constant exchange rates, which is the ultimate measure of channel effectiveness.

Strategy: Review Q4 2025 patient enrollment rates against the 10 new launch country target by end of next week.

Amicus Therapeutics, Inc. (FOLD) - Canvas Business Model: Customer Segments

You're looking at the core groups Amicus Therapeutics, Inc. (FOLD) targets with its specialized rare disease portfolio. Honestly, in this space, the customer isn't just the patient; it's the entire ecosystem that gets the drug to them and pays for it.

Fabry Disease Patients with Amenable Mutations (Galafold)

This segment is defined by a specific genetic characteristic, making it a precision medicine target. Amicus Therapeutics, Inc. (FOLD) estimates that globally, approximately 35 to 50 percent of people living with Fabry disease may have amenable GAL variants. The company is actively trying to find more of these patients; for instance, research suggests there could be over 100,000 undiagnosed people living with Fabry disease globally. The existing patient base on Galafold was about 2,730 patients as of the end of 2024. This therapy maintains a strong market position, capturing 69% of the treated amenable patient market as of Q2/Q3 2025. You can see the revenue growth reflecting this segment's value:

Period	Galafold Net Product Sales
Q1 2025	$104.2 million
Q2 2025	$128.9 million
Q3 2025	$138.3 million

The therapy is approved in more than 40 countries, including the U.S., E.U., U.K., and Japan.

Adult Patients with Late-Onset Pompe Disease (Pombiliti + Opfolda)

This group consists of adult patients with late-onset Pompe disease (LOPD) weighing $\ge$40 kg who are not improving on their current Enzyme Replacement Therapy (ERT) in the U.S.. This is a rapidly growing segment for Amicus Therapeutics, Inc. (FOLD). By the end of 2024, approximately 220 patients were treated or scheduled with the commercial product. The momentum is clear in the sales figures:

Period	Pombiliti + Opfolda Net Product Sales
2024 Full Year	$70.3 million
Q1 2025	$21.0 million
Q2 2025	$25.8 million
Q3 2025	$30.7 million
First Nine Months of 2025	$77.5 million

The company is driving adoption through geographic expansion, with regulatory approvals secured in key markets like Japan in June 2025.

Rare Disease Specialists (Nephrologists, Geneticists, Cardiologists)

These are the key prescribers and influencers who manage the care pathways for patients with Fabry and Pompe diseases. They are the gatekeepers to patient access. Amicus Therapeutics, Inc. (FOLD) focuses its commercial execution efforts directly on this specialized medical community.

• Specialists are targeted based on their patient panels for Fabry disease, where genetic testing is key for Galafold eligibility.
• For Pompe disease, the focus includes specialists managing patients who are not responding adequately to prior ERT regimens.
• The company is expanding its reach, with Pombiliti + Opfolda now approved in the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan as of mid-2025.
• The Netherlands, which has over 150 individuals with Pompe disease, secured a five-year agreement for broad access.

Global Payers and Government Reimbursement Agencies

Securing favorable coverage decisions from payers is critical for commercial success, especially in markets outside the U.S. This segment dictates the net realized price and patient access. Amicus Therapeutics, Inc. (FOLD) has been making significant progress here, which directly impacts revenue projections.

• Reimbursement for Pombiliti + Opfolda is now secured in 15 countries as of Q3 2025.
• Agreements were completed in late 2024 and early 2025 for countries including Italy, Sweden, Switzerland, and the Czech Republic.
• The company anticipated new regulatory decisions in Australia, Canada, and Japan during 2025.
• The U.S. market protection for Galafold is strong, with a settlement preventing generic competition until January 2037.

Amicus Therapeutics, Inc. (FOLD) - Canvas Business Model: Cost Structure

You're looking at the expense side of Amicus Therapeutics, Inc.'s (FOLD) operations as of late 2025. The cost structure is heavily weighted toward supporting global commercial reach and pipeline advancement, which is typical for a company scaling its rare disease portfolio.

The primary cost focus for the full fiscal year 2025 is the projected non-GAAP Operating Expenses, which Amicus Therapeutics guides to be between $380M to $400M. This range reflects the investment needed to support the continued global rollout of their commercial products and ongoing pipeline activities. For context on recent quarterly spend, total GAAP operating expenses were $115.3 million for the third quarter of 2025, up 8% year-over-year, and $148.9 million for the second quarter of 2025, which included a significant one-time cost.

A major component driving the expense structure is the investment in the pipeline, specifically the strategic move into Focal Segmental Glomerulosclerosis (FSGS). This includes costs associated with the exclusive U.S. licensing agreement for DMX-200. The full-year 2025 non-GAAP Operating Expenses guidance explicitly includes a $30M Upfront License Payment for this asset. The Phase 3 study for DMX-200, the ACTION3 study, remains on track for full enrollment by the end of 2025, representing an ongoing commitment to clinical development, even though the study is funded and executed by Dimerix.

Global commercialization and the necessary Selling, General, and Administrative (SG&A) functions represent a substantial fixed and variable cost base. These costs cover the sales force, marketing, and general corporate overhead required to support the growth of Galafold and Pombiliti + Opfolda across international markets. To give you a snapshot of the SG&A component within the GAAP operating expenses, the share-based compensation portion alone for the first quarter of 2025 was reported at $21,168 thousand.

On the revenue side, which offsets these costs, the company maintains a highly favorable cost of goods sold structure. Amicus Therapeutics expects to maintain its Gross Margin in the mid-80% range for 2025. Here is a quick look at the key financial targets influencing the cost absorption:

Metric	2025 Projection/Range
Projected Full Year Non-GAAP Operating Expenses	$380M to $400M
Included DMX-200 Upfront License Payment	$30M
Expected Gross Margin	Mid 80%
Q3 2025 GAAP Operating Expenses	$115.3 million
Q1 2025 SG&A (Share-based Comp. Only)	$21,168 thousand

The cost structure is clearly geared toward maximizing the profitability of its core commercial products while making strategic, upfront investments in late-stage pipeline assets like DMX-200. The goal is to convert the high gross margin product sales into positive GAAP net income, which is anticipated during the second half of 2025.

Amicus Therapeutics, Inc. (FOLD) - Canvas Business Model: Revenue Streams

You're looking at the core engine driving Amicus Therapeutics, Inc.'s financial results, which centers entirely on net product sales from its commercial portfolio. This is where the rubber meets the road for the company's rare disease strategy.

Net product sales from Galafold, the oral precision medicine for Fabry disease, remains the primary revenue driver. For the third quarter of 2025, Galafold generated net product sales of $\text{138.3 million}$ dollars. That represents a year-over-year increase of $\text{15\%}$ reported, or $\text{12\%}$ growth when measured at constant exchange rates (CER). Honestly, the continued execution in all markets and strong patient compliance are keeping this revenue stream robust.

Next up, you see the accelerating net product sales from the Pombiliti + Opfolda launches, which are clearly gaining traction in the Pompe disease market. These two-component therapy sales hit $\text{30.7 million}$ dollars in Q3 2025. The growth rate here is what really stands out; sales jumped $\text{45\%}$ reported year-over-year, or $\text{42\%}$ at CER, driven by high commercial demand and new launch countries. This product line is showing the kind of rapid scaling you want to see from a newer launch.

Here's the quick math on how those two products stacked up in the third quarter of 2025:

Revenue Stream Component	Q3 2025 Net Product Sales (USD)	Year-over-Year Growth (Reported)
Galafold	$\text{138,347,000}$	$\text{15\%}$
Pombiliti + Opfolda	$\text{30,714,000}$	$\text{45\%}$
Total Net Product Revenues	$\text{169,061,000}$	$\text{19\%}$

Looking ahead, Amicus Therapeutics reiterated its guidance for total revenue growth projected at 15%-22% for the full year 2025, measured at constant exchange rates (CER). This range reflects the ongoing momentum from both key products. For context, the year-to-date total revenue through the first nine months of 2025 reached $\text{448.998 million}$ dollars.

A major financial milestone for the revenue story is the achievement of profitability. Amicus Therapeutics achieved GAAP Net Income of $\text{17.3 million}$ dollars in Q3 2025. This was the first quarter in 2025 where the company delivered positive GAAP net income, signaling a transition to a self-sustainable business model.

The revenue streams are supported by key operational metrics:

• Galafold sales growth at CER for the full year 2025 is still projected between $\text{10\%}$ and $\text{15\%}$.
• Pombiliti + Opfolda revenue growth for the full year 2025 is projected between $\text{50\%}$ and $\text{65\%}$ at CER.
• The company expects to surpass $\text{1 billion}$ dollars in total annual sales by 2028.

Finance: draft the Q4 2025 revenue forecast reconciliation by end of next week.