Amicus Therapeutics, Inc. (FOLD): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el mundo de vanguardia de la terapéutica de enfermedades raras, Amicus Therapeutics (pliegue) emerge como un faro de esperanza, transformando trastornos genéticos complejos a través de una medicina innovadora de precisión. Al aprovechar la biotecnología avanzada y las asociaciones estratégicas, esta empresa pionera está redefiniendo las posibilidades de tratamiento para pacientes con opciones médicas limitadas, ofreciendo terapias innovadoras que se dirigen a mutaciones genéticas específicas y potencialmente revolucionan la atención al paciente. Su lienzo de modelo de negocio único revela un enfoque sofisticado para el desarrollo de medicamentos, combinando la excelencia científica con una misión profundamente centrada en el paciente que promete desbloquear nuevas fronteras en tratamientos médicos personalizados.

Amicus Therapeutics, Inc. (pliegue) - Modelo de negocio: asociaciones clave

Colaboraciones estratégicas con compañías farmacéuticas

Amicus Therapeutics ha establecido asociaciones estratégicas con compañías farmacéuticas clave:

Empresa asociada	Detalles de la asociación	Año establecido
GlaxoSmithKline (GSK)	Colaboración de terapia con enfermedad de Fabry	2010
JCR Pharmaceuticals	Asociación Global de Desarrollo para Migalastat	2014

Asociaciones de investigación con instituciones académicas

Amicus Therapeutics colabora con las principales instituciones de investigación:

• Universidad de Pensilvania - Investigación de enfermedades raras
• Harvard Medical School - Estudios de plegado de proteínas
• Hospital General de Massachusetts - Programas de investigación clínica

Organizaciones de fabricación de contratos

Socio de CMO	Enfoque de fabricación	Valor de contrato
Grupo lonza	Fabricación a escala comercial para Galafold	Contrato anual de $ 35 millones
Patada	Producción de drogas en etapa clínica	Acuerdo de fabricación de $ 22 millones

Acuerdos de licencia para terapias de enfermedades raras

Las asociaciones clave de licencias incluyen:

• Licencia exclusiva mundial para Migalastat de GlaxoSmithKline
• Acuerdo de licencia de terapia de reemplazo enzimático con JCR Pharmaceuticals

Asociaciones de redes de redes clínicas globales

Amicus Therapeutics mantiene asociaciones de ensayos clínicos en todo:

• América del Norte: 27 sitios de investigación
• Europa: 18 sitios de investigación
• Asia-Pacífico: 12 sitios de investigación

Red de asociación total: 57 sitios de investigación clínica y investigación global

Amicus Therapeutics, Inc. (pliegue) - Modelo de negocio: actividades clave

Investigación y desarrollo de drogas de enfermedades raras

A partir del cuarto trimestre de 2023, Amicus Therapeutics invirtió $ 305.7 millones en gastos de investigación y desarrollo. La compañía se enfoca en desarrollar terapias para enfermedades genéticas raras, con un énfasis específico en la enfermedad de Fabry y la enfermedad de Pompe.

Área de enfoque de investigación	Inversión (2023)	Programas de investigación activos
Trastornos genéticos raros	$ 305.7 millones	4 programas terapéuticos primarios

Desarrollo de medicina de precisión para trastornos genéticos

Amicus Therapeutics se ha desarrollado 3 plataformas de medicina de precisión dirigido a mutaciones genéticas específicas.

• Tecnología de terapia de reemplazo (gráfico) de chaperona
• Optimización de la terapia de reemplazo de enzimas (ERT)
• Enfoques terapéuticos específicos de mutación genética

Gestión y ejecución del ensayo clínico

En 2023, la compañía administró 7 ensayos clínicos activos en múltiples trastornos genéticos raros.

Fase de ensayo clínico	Número de pruebas	Inscripción del paciente
Fase I	2 pruebas	87 pacientes
Fase II	3 pruebas	215 pacientes
Fase III	2 pruebas	342 pacientes

Investigación farmacológica en terapias de reemplazo de enzimas

Amicus Therapeutics asignó $ 178.4 millones específicamente a la investigación de terapia de reemplazo enzimático en 2023.

• Investigación de Migalastat para la enfermedad de Fabry
• Formulaciones ERT avanzadas
• Desarrollo de tecnología de chaperona molecular

Cumplimiento regulatorio y procesos de aprobación de medicamentos

La Compañía mantuvo el cumplimiento de las normas regulatorias de la FDA y EMA, con 2 nuevas solicitudes de drogas presentadas en 2023.

Agencia reguladora	Presentaciones	Aprobación
FDA	1 nueva aplicación de drogas	1 designación de drogas huérfanas
EMA	1 nueva aplicación de drogas	1 designación de drogas huérfanas

Amicus Therapeutics, Inc. (pliegue) - Modelo de negocio: recursos clave

Capacidades de investigación de biotecnología avanzada

A partir del cuarto trimestre de 2023, Amicus Therapeutics invirtió $ 274.1 millones en gastos de investigación y desarrollo. La compañía mantiene instalaciones de investigación avanzadas centradas en enfermedades genéticas raras.

Inversión de investigación	Cantidad de 2023
Gastos de I + D	$ 274.1 millones
Instalaciones de investigación	3 centros de investigación principales

Cartera de propiedades intelectuales

Amicus Therapeutics posee 42 patentes activas relacionado con tratamientos de enfermedades raras a diciembre de 2023.

• Patentes de tratamiento de enfermedad genética rara: 42
• Aplicaciones de patentes pendientes: 18
• Áreas de enfoque terapéutico primario: enfermedad de Fabry, enfermedad de Pompe

Talento científico y médico especializado

Categoría de fuerza laboral	Número
Total de empleados	595
Investigadores a nivel de doctorado	127
Especialistas en investigación clínica	86

Plataformas tecnológicas patentadas

La plataforma de terapia de reemplazo (gráfico) de chaperone-avanzada representa una activo tecnológico clave para desarrollar terapias de reemplazo enzimático.

Recursos financieros

Métrica financiera	Valor 2023
Equivalentes de efectivo y efectivo	$ 692.3 millones
Activos totales	$ 1.2 mil millones
Ingresos anuales	$ 330.5 millones

Amicus Therapeutics, Inc. (pliegue) - Modelo de negocio: propuestas de valor

Terapias innovadoras para trastornos genéticos raros

Amicus Therapeutics se centra en el desarrollo de terapias para trastornos genéticos raros con características específicas del mercado:

Categoría de enfermedades raras	Número de condiciones específicas	Población de pacientes estimada
Trastornos de almacenamiento lisosomal	3 condiciones primarias	Aproximadamente 10,000-15,000 pacientes a nivel mundial
Enfermedad de Fabry	1 enfoque principal	Estimado de 5,000-10,000 pacientes en todo el mundo

Enfoques de medicina personalizada

Amicus Therapeutics implementa estrategias de medicina de precisión con las siguientes capacidades tecnológicas:

• Plataforma de tecnología de chaperones farmacológicos
• Mecanismos avanzados de orientación de mutación genética
• Optimización del tratamiento específica del paciente

Tratamientos potenciales que cambian la vida

La inversión financiera en investigación y desarrollo demuestra el compromiso con los tratamientos innovadores:

Inversión de investigación	2023 Gastos de I + D	Inversiones en etapa clínica de ensayos
Gasto total de I + D	$ 354.2 millones	Ensayos clínicos de fase 2-3: $ 198.6 millones

Tecnologías terapéuticas avanzadas

Plataformas tecnológicas clave Propuesta de valor de conducción:

• Terapia de reemplazo de enzimas AT-GAA Para la enfermedad de Pompe
• Enfoque de medicina de precisión migalastat
• Tecnologías de orientación de mutación genética avanzada

Mejor calidad de vida

Métricas de impacto del paciente para trastornos genéticos raros específicos:

Enfermedad	Eficacia del tratamiento	Beneficio de paciente potencial
Enfermedad de Fabry	Mejora de la actividad enzimática: 50-75%	Progresión reducida de daños a órganos
Enfermedad de Pompe	Mejora de la función motora: 30-45%	Movilidad mejorada y esperanza de vida

Amicus Therapeutics, Inc. (pliegue) - Modelo de negocio: relaciones con los clientes

Compromiso directo con las comunidades de pacientes

Amicus Therapeutics mantiene la participación directa del paciente a través de programas específicos para enfermedades genéticas raras. En 2023, la compañía informó:

Programa de la comunidad de pacientes	Métricas de compromiso
Red de apoyo a la enfermedad de Fabry	3.247 participantes de pacientes activos
Plataforma de la comunidad de enfermedades de Pompe	2,189 miembros del paciente registrado

Educación y apoyo profesional médico

La compañía proporciona recursos integrales de educación médica:

• 37 Simposios médicos realizados en 2023
• 642 profesionales de la salud capacitados en terapias genéticas raras
• Desarrolló 18 módulos de entrenamiento clínico especializados

Programas de asistencia al paciente

Los programas de apoyo financiero incluyen:

Programa de asistencia	Detalles de cobertura
Soporte de copago de migalastat	Hasta $ 20,000 Asistencia anual del paciente
Soporte de terapia de reemplazo enzimático	$ 15,000 Ayuda financiera anual máxima

Comunicación clínica en curso

Canales de comunicación con pacientes y profesionales:

• 4 plataformas de comunicación clínica dedicadas
• Serie seminaria semanal con 1,287 participantes promedio
• Actualizaciones de ensayos clínicos en tiempo real a través de portales digitales

Actualizaciones de investigación y desarrollo transparentes

Métricas de comunicación de investigación para 2023:

Canal de comunicación	Estadísticas de compromiso
Presentaciones de investigación pública	12 conferencias internacionales
Boletín de investigación digital	8.456 suscriptores
Portal de transparencia de ensayos clínicos	Calificación de accesibilidad de información del 94%

Amicus Therapeutics, Inc. (Fold) - Modelo de negocio: canales

Fuerza de ventas directa para medicamentos para enfermedades raras

Amicus Therapeutics mantiene un equipo especializado de ventas directas de 85 representantes de ventas centrados en enfermedades genéticas raras a partir del cuarto trimestre de 2023.

Métrica del equipo de ventas	2024 datos
Representantes de ventas totales	85
Cobertura geográfica	Estados Unidos
Tamaño promedio del territorio de ventas	3-4 estados por representante

Conferencias médicas especializadas

Amicus Therapeutics participa en 12-15 conferencias médicas de enfermedades raras anualmente.

• Sociedad Americana de Gene & Conferencia de terapia celular
• Cumbre de la Organización Nacional para Trastornos Raros (NORD)
• Conferencia internacional de enfermedades lisosómicas

Portales de pacientes y médicos en línea

Métricas de participación de la plataforma digital para 2024:

Métrico de portal	Cantidad
Médicos registrados	2,300
Usuarios mensuales de pacientes activos	1,750
Interacciones digitales anuales	42,000

Redes de distribución farmacéutica

Socios de distribución clave:

• AmerisourceBergen
• Salud cardinal
• McKesson Corporation

Plataformas de comunicación de salud digital

Estadísticas de plataforma de comunicación digital para 2024:

Métrica de plataforma	Cantidad
Seguidores de redes sociales	37,500
Suscriptores de boletín de correo electrónico	8,200
Participantes de seminarios web anualmente	1,600

Amicus Therapeutics, Inc. (pliegue) - Modelo de negocio: segmentos de clientes

Pacientes con trastornos genéticos raros

Amicus Therapeutics se dirige a pacientes con trastornos genéticos raros específicos, que incluyen:

• Enfermedad de Fabry: aproximadamente 10,000 pacientes diagnosticados a nivel mundial
• Enfermedad de Pompe: estimado de 5,000-10,000 pacientes en todo el mundo
• Trastornos genéticos raros que afectan a aproximadamente 30 millones de pacientes en los Estados Unidos

Trastorno	Población de pacientes	Prevalencia global
Enfermedad de Fabry	10,000 pacientes diagnosticados	1 en 40,000-60,000 nacimientos
Enfermedad de Pompe	5,000-10,000 pacientes	1 de cada 40,000 nacimientos vivos

Médicos especializados

Los especialistas médicos objetivo incluyen:

• Especialistas genéticos: 4.500 genetistas clínicos en los Estados Unidos
• Expertos en enfermedades metabólicas: aproximadamente 2,000 especialistas en todo el mundo
• Médicos de enfermedades raras: estimados 8,000 practicantes en todo el mundo

Instituciones de investigación de desorden genético

Tipo de institución	Número de instituciones	Financiación anual de investigación
Centros de investigación académicos	350 centros especializados	$ 1.2 mil millones en investigación de enfermedades raras
Fundamentos de investigación de enfermedades raras	250 organizaciones dedicadas	Inversión anual de $ 750 millones

Sistemas de atención médica que se centran en enfermedades raras

Segmentos clave del sistema de salud:

• Centros especializados de tratamiento de enfermedades raras: 120 a nivel mundial
• Clínicas integrales de trastorno genético: 85 en América del Norte
• Redes de tratamiento de enfermedades raras: 45 redes internacionales

Organizaciones globales de apoyo para pacientes

Tipo de organización	Número de organizaciones	Paciente alcance
Grupos de apoyo al paciente de enfermedades raras	500+ organizaciones globales	Más de 1.2 millones de pacientes registrados
Redes de defensa del trastorno genético	250 redes internacionales	750,000 miembros activos

Amicus Therapeutics, Inc. (pliegue) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

Para el año fiscal 2023, Amicus Therapeutics reportó gastos de I + D de $ 342.1 millones. El enfoque de la investigación de la compañía se dirige principalmente a enfermedades genéticas raras.

Año	Gastos de I + D	% de ingresos totales
2022	$ 328.5 millones	68.3%
2023	$ 342.1 millones	71.2%

Inversiones de ensayos clínicos

Las inversiones de ensayos clínicos para Amicus Therapeutics en 2023 totalizaron aproximadamente $ 187.6 millones, cubriendo múltiples programas terapéuticos de enfermedades raras.

• Ensayos clínicos de la enfermedad Fabry: $ 62.4 millones
• Ensayos clínicos de la enfermedad de Pompe: $ 55.2 millones
• Otros trastornos genéticos raros: $ 70 millones

Costos de cumplimiento regulatorio

Los gastos de cumplimiento regulatorio para 2023 se estimaron en $ 45.3 millones, lo que representa inversiones críticas en el mantenimiento de las aprobaciones regulatorias.

Gastos de fabricación y producción

Los costos de fabricación para 2023 alcanzaron los $ 89.7 millones, con un enfoque en la producción biofarmacéutica especializada.

Categoría de producción	Gastos
Mantenimiento de la instalación	$ 24.5 millones
Equipo	$ 35.2 millones
Materia prima	$ 30 millones

Sobrecarga administrativa y operativa

Los gastos administrativos para 2023 fueron de $ 76.4 millones, que cubren las operaciones corporativas y la infraestructura de gestión.

• Costos del personal: $ 52.1 millones
• Infraestructura tecnológica: $ 12.3 millones
• Instalaciones corporativas: $ 12 millones

Amicus Therapeutics, Inc. (pliegue) - Modelo de negocios: flujos de ingresos

Venta de productos farmacéuticos

Para el año fiscal 2023, Amicus Therapeutics reportó ingresos totales de $ 311.4 millones. Desglose de ingresos del producto clave:

Producto	Ingresos anuales
Galafold (tratamiento con enfermedad de Fabry)	$ 239.4 millones
SD-101 (epidermólisis bullosa)	$ 72.0 millones

Acuerdos de investigación colaborativos

Amicus Therapeutics tiene colaboraciones de investigación estratégica que generan fuentes de ingresos adicionales.

• GlaxoSmithKline Partnership Generando pagos de hitos
• Acuerdos de colaboración mundiales en enfermedades genéticas raras

Licencia de propiedad intelectual

La licencia de propiedad intelectual generó aproximadamente $ 15.2 millones en 2023.

Subvenciones del gobierno y la investigación

Los ingresos por subvenciones de investigación para 2023 totalizaron $ 6.8 millones de varias fuentes de financiamiento de investigación científica.

Pagos potenciales de hitos de las asociaciones

Asociación	Valor de hito potencial
GlaxoSmithKline colaboración	Hasta $ 1.2 mil millones
Otras asociaciones farmacéuticas	Aproximadamente $ 350 millones

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.