Amicus Therapeutics, Inc. (pli): Business Model Canvas [Jan-2025 Mis à jour]

Dans le monde de pointe de la thérapeutique des maladies rares, Amicus Therapeutics (pli) apparaît comme un phare d'espoir, transformant des troubles génétiques complexes grâce à une médecine de précision innovante. En tirant parti de la biotechnologie avancée et des partenariats stratégiques, cette entreprise pionnière redéfinit les possibilités de traitement pour les patients avec des options médicales limitées, offrant des thérapies révolutionnaires qui ciblent des mutations génétiques spécifiques et potentiellement révolutionner les soins aux patients. Leur toile de modèle commercial unique révèle une approche sophistiquée du développement de médicaments, combinant l'excellence scientifique avec une mission profondément centrée sur le patient qui promet de débloquer de nouvelles frontières dans les traitements médicaux personnalisés.

Amicus Therapeutics, Inc. (pli) - Modèle commercial: partenariats clés

Collaborations stratégiques avec les sociétés pharmaceutiques

Amicus Therapeutics a établi des partenariats stratégiques avec des sociétés pharmaceutiques clés:

Entreprise partenaire	Détails du partenariat	Année établie
GlaxoSmithKline (GSK)	Collaboration de la thérapie de la maladie de Fabry	2010
Jcr pharmaceuticals	Partenariat de développement mondial pour migalastat	2014

Partenariats de recherche avec les établissements universitaires

Amicus Therapeutics collabore avec les principaux institutions de recherche:

• Université de Pennsylvanie - Recherche de maladies rares
• École de médecine de Harvard - Études de pliage des protéines
• Hôpital général du Massachusetts - Programmes de recherche clinique

Organisations de fabrication de contrats

Partenaire CMO	Focus de la fabrication	Valeur du contrat
Groupe Lonza	Fabrication à l'échelle commerciale pour Galafold	Contrat annuel de 35 millions de dollars
Patheron	Production de médicaments à stade clinique	Contrat de fabrication de 22 millions de dollars

Accords de licence pour les thérapies par maladies rares

Les partenariats clés de licence comprennent:

• Licence exclusive mondiale pour Migalastat de GlaxoSmithKline
• Contrat de licence de thérapie de remplacement enzymatique avec JCR Pharmaceuticals

Partenariats du réseau mondial des essais cliniques

Amicus Therapeutics maintient des partenariats d'essais cliniques à travers:

• Amérique du Nord: 27 sites de recherche
• Europe: 18 sites de recherche
• Asie-Pacifique: 12 sites de recherche

Réseau total de partenariat: 57 sites mondiaux de recherche et d'essais cliniques

Amicus Therapeutics, Inc. (pli) - Modèle commercial: activités clés

Recherche et développement de médicaments contre les maladies rares

Au quatrième trimestre 2023, Amicus Therapeutics a investi 305,7 millions de dollars dans les frais de recherche et développement. L'entreprise se concentre sur le développement de thérapies pour les maladies génétiques rares, avec un accent spécifique sur la maladie de Fabry et la maladie de Pompe.

Domaine de mise au point de recherche	Investissement (2023)	Programmes de recherche actifs
Troubles génétiques rares	305,7 millions de dollars	4 programmes thérapeutiques primaires

Développement de la médecine de précision pour les troubles génétiques

Amicus Therapeutics a développé 3 plateformes de médecine de précision ciblant des mutations génétiques spécifiques.

• Technologie de thérapie de remplacement (graphique) avancé par chaperon
• Optimisation de la thérapie de remplacement enzymatique (ERT)
• Approches thérapeutiques spécifiques à la mutation génétique

Gestion et exécution des essais cliniques

En 2023, la société a géré 7 essais cliniques actifs sur plusieurs troubles génétiques rares.

Phase d'essai clinique	Nombre de procès	Inscription des patients
Phase I	2 essais	87 patients
Phase II	3 essais	215 patients
Phase III	2 essais	342 patients

Recherche pharmacologique dans les thérapies de remplacement des enzymes

Amicus Therapeutics a alloué 178,4 millions de dollars spécifiquement à la recherche sur la thérapie de remplacement enzymatique en 2023.

• Recherche de migalastat pour la maladie de Fabry
• Formulations ERT avancées
• Développement de la technologie du chaperon moléculaire

Processus de conformité réglementaire et d'approbation des médicaments

La société a maintenu le respect des normes réglementaires de la FDA et de l'EMA, avec 2 nouvelles demandes de médicament soumises en 2023.

Agence de réglementation	Soumissions	Approbations
FDA	1 nouvelle demande de médicament	1 désignation de médicament orphelin
Ema	1 nouvelle demande de médicament	1 désignation de médicament orphelin

Amicus Therapeutics, Inc. (pli) - Modèle commercial: Ressources clés

Capacités de recherche avancées de la biotechnologie

Au quatrième trimestre 2023, Amicus Therapeutics a investi 274,1 millions de dollars dans les frais de recherche et développement. La société maintient des installations de recherche avancées axées sur les maladies génétiques rares.

Investissement en recherche	2023 Montant
Dépenses de R&D	274,1 millions de dollars
Installations de recherche	3 centres de recherche primaires

Portefeuille de propriété intellectuelle

Amicus Therapeutics tient 42 brevets actifs lié à des traitements de maladies rares en décembre 2023.

• Brevets de traitement des maladies génétiques rares: 42
• Demandes de brevet en instance: 18
• Primaires des domaines de mise au point thérapeutique: maladie de Fabry, maladie de Pompe

Talent scientifique et médical spécialisé

Catégorie de main-d'œuvre	Nombre
Total des employés	595
Chercheurs au niveau du doctorat	127
Spécialistes de la recherche clinique	86

Plateformes technologiques propriétaires

La plate-forme de thérapie de remplacement (graphique) révolue par chaperon représente une atout technologique clé pour développer des thérapies de remplacement enzymatiques.

Ressources financières

Métrique financière	Valeur 2023
Equivalents en espèces et en espèces	692,3 millions de dollars
Actif total	1,2 milliard de dollars
Revenus annuels	330,5 millions de dollars

Amicus Therapeutics, Inc. (pli) - Modèle d'entreprise: propositions de valeur

Thérapies innovantes pour les troubles génétiques rares

Amicus Therapeutics se concentre sur le développement de thérapies pour des troubles génétiques rares avec des caractéristiques spécifiques du marché:

Catégorie de maladies rares	Nombre de conditions ciblées	Population estimée des patients
Troubles du stockage lysosomal	3 conditions primaires	Environ 10 000 à 15 000 patients dans le monde
Maladie de Fabry	1 Focus primaire	Estimé 5 000 à 10 000 patients dans le monde

Approches de médecine personnalisée

Amicus Therapeutics implémente les stratégies de médecine de précision avec les capacités technologiques suivantes:

• Plateforme de technologie de chaperon pharmacologique
• Mécanismes de ciblage de mutation génétique avancée
• Optimisation du traitement spécifique au patient

Traitements potentiels qui changent la vie

L'investissement financier dans la recherche et le développement démontre un engagement envers les traitements révolutionnaires:

Investissement en recherche	2023 dépenses de R&D	Investissements en phase d'essai clinique
Dépenses totales de R&D	354,2 millions de dollars	Phase 2-3 essais cliniques: 198,6 millions de dollars

Technologies thérapeutiques avancées

Plateformes technologiques clés conduisant la proposition de valeur:

• Thérapie de remplacement de l'enzyme AT-GAA pour la maladie de Pompe
• Approche de médecine de précision de la précision migalastat
• Technologies de ciblage de mutation génétique avancée

Amélioration de la qualité de vie

Les mesures d'impact des patients pour les troubles génétiques rares ciblés:

Maladie	Efficacité du traitement	Avantage potentiel du patient
Maladie de Fabry	Amélioration de l'activité enzymatique: 50-75%	Réduction de la progression des dommages aux organes
Maladie de Pompe	Amélioration de la fonction motrice: 30-45%	Mobilité améliorée et espérance de vie

Amicus Therapeutics, Inc. (pli) - Modèle d'entreprise: relations clients

Engagement direct avec les communautés de patients

Amicus Therapeutics maintient l'engagement direct des patients grâce à des programmes ciblés pour des maladies génétiques rares. En 2023, la société a rapporté:

Programme communautaire des patients	Métriques d'engagement
Réseau de soutien à la maladie de Fabry	3 247 participants aux patients actifs
Plateforme communautaire de la maladie de Pompe	2 189 membres du patient enregistré

Éducation et soutien professionnel de la santé

L'entreprise propose des ressources complètes de formation médicale:

• 37 Symposiaux médicaux réalisés en 2023
• 642 professionnels de la santé formés sur des thérapies génétiques rares
• Développé 18 modules de formation clinique spécialisés

Programmes d'aide aux patients

Les programmes de soutien financier comprennent:

Programme d'assistance	Détails de la couverture
Support de copaiement migalastat	Jusqu'à 20 000 $ d'aide annuelle aux patients
Support de thérapie de remplacement enzymatique	15 000 $ Aide financière annuelle maximale

Communication clinique en cours

Canaux de communication avec les patients et les professionnels:

• 4 plateformes de communication cliniques dédiées
• Série de webinaires hebdomadaires avec 1 287 participants moyens
• Mises à jour des essais cliniques en temps réel via des portails numériques

Mises à jour transparentes de la recherche et du développement

Métriques de communication de recherche pour 2023:

Canal de communication	Statistiques d'engagement
Présentations de recherche publique	12 conférences internationales
Newsletter de recherche numérique	8 456 abonnés
Portail de transparence des essais cliniques	Évaluation de l'accessibilité à 94%

Amicus Therapeutics, Inc. (pli) - Modèle d'entreprise: canaux

Force de vente directe pour les médicaments contre les maladies rares

Amicus Therapeutics maintient une équipe de vente directe spécialisée de 85 représentants des ventes axés sur les maladies génétiques rares au quatrième trimestre 2023.

Métrique de l'équipe de vente	2024 données
Représentants des ventes totales	85
Couverture géographique	États-Unis
Taille moyenne du territoire des ventes	3-4 États par représentant

Conférences médicales spécialisées

Amicus Therapeutics participe à 12 à 15 conférences médicales de maladies rares chaque année.

• Société américaine de gène & Conférence de thérapie cellulaire
• Sommet national de l'Organisation des troubles rares (NORD)
• Conférence internationale des maladies lysosomales

Portails de patients et de médecins en ligne

Métriques d'engagement de la plate-forme numérique pour 2024:

Métrique du portail	Quantité
Médecins enregistrés	2,300
Utilisateurs mensuels des patients actifs	1,750
Interactions numériques annuelles	42,000

Réseaux de distribution pharmaceutique

Partenaires de distribution clés:

• Amerisourcebergen
• Santé cardinale
• McKesson Corporation

Plateformes de communication de santé numérique

Statistiques de la plate-forme de communication numérique pour 2024:

Métrique de la plate-forme	Quantité
Abonnés des médias sociaux	37,500
Envoyez un e-mail aux abonnés à la newsletter	8,200
Participants au webinaire chaque année	1,600

Amicus Therapeutics, Inc. (pli) - Modèle d'entreprise: segments de clientèle

Patients souffrant de troubles génétiques rares

Amicus Therapeutics cible les patients souffrant de troubles génétiques rares spécifiques, notamment:

• Maladie de Fabry: environ 10 000 patients diagnostiqués dans le monde
• Maladie Pompe: 5 000 à 10 000 patients dans le monde
• Des troubles génétiques rares affectant environ 30 millions de patients aux États-Unis

Trouble	Population de patients	Prévalence mondiale
Maladie de Fabry	10 000 patients diagnostiqués	1 naissance sur 40 000 à 60 000
Maladie de Pompe	5 000 à 10 000 patients	1 naissances vivantes sur 40 000

Médecins spécialisés

Les médecins spécialistes cibles comprennent:

• Spécialistes génétiques: 4 500 généticiens cliniques aux États-Unis
• Experts en maladie métabolique: environ 2 000 spécialistes du monde entier
• Médecins rares: estimé 8 000 praticiens dans le monde

Institutions de recherche sur les troubles génétiques

Type d'institution	Nombre d'institutions	Financement de la recherche annuelle
Centres de recherche universitaires	350 centres spécialisés	1,2 milliard de dollars en recherche de maladies rares
Fondations de recherche sur les maladies rares	250 organisations dédiées	Investissement annuel de 750 millions de dollars

Systèmes de soins de santé axés sur les maladies rares

Segments clés du système de soins de santé:

• Centres de traitement des maladies rares spécialisées: 120 dans le monde
• Cliniques complètes des troubles génétiques: 85 en Amérique du Nord
• Réseaux de traitement des maladies rares: 45 réseaux internationaux

Organisations mondiales de soutien aux patients

Type d'organisation	Nombre d'organisations	Patient à portée de patient
Groupes de soutien aux patients atteints de maladies rares	Plus de 500 organisations mondiales	Plus de 1,2 million de patients enregistrés
Réseaux de plaidoyer des troubles génétiques	250 réseaux internationaux	750 000 membres actifs

Amicus Therapeutics, Inc. (pli) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement approfondies

Pour l'exercice 2023, Amicus Therapeutics a déclaré des dépenses de R&D de 342,1 millions de dollars. L'orientation de la recherche de l'entreprise cible principalement les maladies génétiques rares.

Année	Dépenses de R&D	% des revenus totaux
2022	328,5 millions de dollars	68.3%
2023	342,1 millions de dollars	71.2%

Investissements d'essais cliniques

Les investissements en essais cliniques pour Amicus Therapeutics en 2023 ont totalisé environ 187,6 millions de dollars, couvrant plusieurs programmes thérapeutiques de maladies rares.

• Essais cliniques de la maladie de Fabry: 62,4 millions de dollars
• Essais cliniques de la maladie de Pompe: 55,2 millions de dollars
• Autres troubles génétiques rares: 70 millions de dollars

Coûts de conformité réglementaire

Les frais de conformité réglementaire pour 2023 étaient estimés à 45,3 millions de dollars, ce qui représente les investissements critiques dans le maintien des approbations réglementaires.

Frais de fabrication et de production

Les coûts de fabrication pour 2023 ont atteint 89,7 millions de dollars, en mettant l'accent sur la production biopharmaceutique spécialisée.

Catégorie de production	Frais
Entretien d'installation	24,5 millions de dollars
Équipement	35,2 millions de dollars
Matières premières	30 millions de dollars

Surfaçon administratives et opérationnelles

Les frais administratifs pour 2023 étaient de 76,4 millions de dollars, couvrant les opérations des entreprises et les infrastructures de gestion.

• Coûts de personnel: 52,1 millions de dollars
• Infrastructure technologique: 12,3 millions de dollars
• Facilités d'entreprise: 12 millions de dollars

Amicus Therapeutics, Inc. (pli) - Modèle commercial: Strots de revenus

Ventes de produits pharmaceutiques

Pour l'exercice 2023, Amicus Therapeutics a déclaré un chiffre d'affaires total de 311,4 millions de dollars. Réflexion des revenus des produits clés:

Produit	Revenus annuels
Galafold (traitement de la maladie de Fabry)	239,4 millions de dollars
SD-101 (épidermolyse bullosa)	72,0 millions de dollars

Accords de recherche collaborative

Amicus Therapeutics a des collaborations de recherche stratégique générant des sources de revenus supplémentaires.

• GlaxoSmithKline Partnership générant des paiements de jalons
• Accords de collaboration mondiaux dans des maladies génétiques rares

Licence de propriété intellectuelle

Les licences de propriété intellectuelle ont généré environ 15,2 millions de dollars en 2023.

Subventions gouvernementales et de recherche

Les revenus de subventions de recherche pour 2023 ont totalisé 6,8 millions de dollars provenant de diverses sources de financement de recherche scientifique.

Payments de jalons potentiels à partir de partenariats

Partenariat	Valeur de jalon potentielle
Collaboration GlaxoSmithKline	Jusqu'à 1,2 milliard de dollars
Autres partenariats pharmaceutiques	Environ 350 millions de dollars

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.