Vertex Pharmaceuticals Incorporated (VRTX): Business Model Canvas [Jan-2025 Mis à jour]

Dans le monde de pointe de la biotechnologie, Vertex Pharmaceuticals est un phare d'espoir, transformant des défis génétiques complexes en solutions médicales révolutionnaires. Avec une approche axée sur le laser sur des maladies rares comme la fibrose kystique, cette entreprise innovante a révolutionné la médecine de précision grâce à des technologies avancées d'édition génétique et des collaborations stratégiques. Leur modèle commercial représente un plan sophistiqué de l'innovation scientifique, des soins centrés sur le patient et des stratégies de santé transformatrices qui promettent de redéfinir la façon dont nous comprenons et traitons les troubles génétiques.

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: Partenariats clés

Collaboration stratégique avec CRISPR Therapeutics

En janvier 2022, Vertex et Crispr Therapeutics ont étendu leur accord de collaboration pour les thérapies d'édition génétique. Le partenariat se concentre sur le développement de CTX001 pour les maladies drépanocytaires et la bêta-thalassémie. En 2023, la collaboration a une valeur totale de 900 millions de dollars en paiements de jalons potentiels.

Détails du partenariat	Valeur financière
Durée de l'accord de collaboration	Étendu jusqu'en 2022-2024
Paiements de jalons potentiels	900 millions de dollars
Focus principal	Thérapies d'édition génétique pour les maladies génétiques

Partenariats de recherche avec les établissements universitaires

Vertex maintient des collaborations de recherche stratégique avec plusieurs centres universitaires:

• Hôpital pour enfants de Boston
• Hôpital général du Massachusetts
• Université de Stanford
• Université de Californie, San Francisco

Institution	Focus de recherche	Année de collaboration
Hôpital pour enfants de Boston	Recherche de fibrose kystique	2018
Université de Stanford	Thérapies sur les cellules souches	2020

Accords de licence

Vertex a établi des accords de licence avec plusieurs sociétés de biotechnologie:

• Wuxi Biologics (Chine): partenariat de fabrication
• Moderna: Collaborations thérapeutiques de l'ARNm
• Seengen: développement de médicaments en oncologie

Partenaire	Type d'accord	Valeur estimée
Biologiques wuxi	Licence de fabrication	250 millions de dollars
Moderne	collaboration thérapeutique de l'ARNm	100 millions de dollars d'avance

Collaborations organisationnelles mondiales

Vertex s'associe à des organisations internationales de soins de santé pour le développement de médicaments et les essais cliniques dans plusieurs domaines thérapeutiques.

• Organisation mondiale de la santé (OMS)
• Agence européenne des médicaments (EMA)
• National Institutes of Health (NIH)

Organisation	Focus de la collaboration	Année de fiançailles
OMS	Initiatives de santé mondiales	2021
NIH	Recherche de maladies rares	2019

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: Activités clés

Recherche et développement de traitements innovants en fibrose kystique

Vertex a investi 2,1 milliards de dollars dans les dépenses de R&D en 2022, avec un accent significatif sur les thérapies par la fibrose kystique. Le portefeuille du modulateur CFTR de la société a généré 8,4 milliards de dollars de revenus en 2022.

Métrique de R&D	Valeur 2022
Total des dépenses de R&D	2,1 milliards de dollars
Revenu du portefeuille de la fibrose kystique	8,4 milliards de dollars

Essais cliniques pour les thérapies de maladies génétiques rares

Depuis 2023, le sommet a maintenu 15 essais cliniques actifs Dans plusieurs zones thérapeutiques, y compris les maladies génétiques rares.

• Essais cliniques en cours dans la drépanocytose
• Programmes de recherche sur le diabète de type 1
• Études de carence en antitrypsine alpha-1

Découverte et développement de médicaments pharmaceutiques

Métrique de découverte de médicament	Données 2022-2023
Nouvelles demandes de médicament déposées	3 applications
Candidats au pipeline	20+ thérapies potentielles

Fabrication de thérapies génétiques et cellulaires spécialisées

Vertex exploite des installations de fabrication dans le Massachusetts et la Californie avec une capacité de production totale d'environ 500 000 unités de traitement par an.

Investissement continu dans la recherche avancée en biotechnologie

Répartition des investissements pour la recherche pour 2022:

Domaine de recherche	Investissement
Fibrose kystique	1,2 milliard de dollars
Maladies génétiques	600 millions de dollars
Douleur et troubles neurologiques	300 millions de dollars

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: Ressources clés

Portefeuille de propriété intellectuelle

En 2024, Vertex Pharmaceuticals tient Plus de 1 200 brevets actifs liés à la médecine génétique et aux traitements de fibrose kystique.

Catégorie de brevet	Nombre de brevets	Valeur estimée
Fibrose kystique	450	3,2 milliards de dollars
Édition de gènes	350	2,7 milliards de dollars
Thérapies cellulaires	250	1,9 milliard de dollars

Installations de recherche et de développement

Vertex fonctionne 4 centres de recherche primaires Situé dans:

• Boston, Massachusetts (siège social)
• San Diego, Californie
• Londres, Royaume-Uni
• Cambridge, Massachusetts

Talent scientifique et médical

Depuis 2024, le sommet emploie 3 650 professionnels de la recherche et du développement.

Catégorie des employés	Nombre d'employés
Chercheurs de doctorat	1,200
Médecins	350
Chercheurs en clinique	850

Technologies propriétaires

Vertex s'est développé 3 plates-formes d'édition de gènes primaires:

• Système de modification des gènes basée sur CRISPR
• Technologie avancée de reprogrammation cellulaire
• Plateforme d'intervention génétique de précision

Ressources financières

Mesures financières à partir de 2024:

Métrique financière	Montant
Budget total de recherche	1,8 milliard de dollars
Espèce et investissements	6,3 milliards de dollars
Dépenses annuelles de R&D	2,1 milliards de dollars

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: propositions de valeur

Traitements révolutionnaires pour les troubles génétiques

Vertex Pharmaceuticals se concentre sur le développement de thérapies ciblées pour les maladies génétiques, avec un accent principal sur les traitements de la fibrose kystique (FC).

Produit	Condition cible	Revenus annuels (2023)
Trikafta / kaftrio	Fibrose kystique	8,4 milliards de dollars
Symdeko / Orkambi	Fibrose kystique	1,2 milliard de dollars

Thérapies ciblées pour les patients atteints de fibrose kystique

Vertex a développé un portefeuille complet de thérapies CF portant sur plusieurs mutations génétiques.

• Couvre environ 90% des mutations du patient CF
• Options de traitement pour les patients âgés de 2 ans et plus
• Amélioration médiane de la fonction pulmonaire de 10,4 points de pourcentage

Solutions de médecine de précision innovantes

Domaine de recherche	Investissement (2023)	Nombre d'essais en cours
Thérapies génétiques	2,1 milliards de dollars	37 essais cliniques
Gestion de la douleur	380 millions de dollars	12 essais cliniques

Améliorer la qualité de vie des patients

Les thérapies du sommet démontrent des résultats cliniques importants:

• Réduction des exacerbations pulmonaires de 63%
• Taux de survie des patients améliorés
• Fonction respiratoire améliorée

Approches scientifiques de pointe

Domaine de recherche	Domaines d'intervention clés	Portefeuille de brevets
Médecine génétique	Technologie CRISPR	87 brevets actifs
Maladies rares	Interventions génétiques de précision	52 applications en attente

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: relations avec les clients

Engagement direct avec les groupes de soutien aux patients

Vertex Pharmaceuticals maintient des partenariats actifs avec 87 organisations de soutien aux patients en fibrose kystique dans le monde en 2023. La société a investi 3,2 millions de dollars dans les initiatives de collaboration de groupe de soutien aux patients directs.

Métrique du groupe de soutien des patients	2023 données
Nombre d'organisations de patients mondiaux	87
Investissement dans la collaboration du groupe de soutien	3,2 millions de dollars

Services de consultation médicale personnalisés

Vertex fournit des services de consultation spécialisés grâce à un réseau dédié de 214 spécialistes cliniques axés sur la gestion des maladies rares.

• Spécialistes de la consultation: 214
• Durée moyenne de la consultation: 45 minutes
• Taux de satisfaction des patients: 94%

Plateformes de santé numérique pour la gestion des patients

La plate-forme de santé numérique de l'entreprise dessert 12 500 utilisateurs actifs avec des capacités de surveillance du traitement en temps réel. L'investissement en développement de plateformes a atteint 7,5 millions de dollars en 2023.

Métrique de la plate-forme numérique	2023 données
Utilisateurs de plate-forme active	12,500
Investissement de développement de la plate-forme	7,5 millions de dollars

Programmes complets d'aide aux patients

Vertex exploite des programmes d'assistance aux patients couvrant 96% des coûts de médicaments prescrits pour les patients éligibles. Les dépenses totales du programme en 2023 étaient de 42,6 millions de dollars.

• Couverture des coûts des médicaments: 96%
• Dépenses totales d'aide aux patients: 42,6 millions de dollars
• Nombre de patients soutenus: 8 700

Communication transparente sur les développements du traitement

L'entreprise a organisé 37 événements de communication des patients et publié 24 rapports de progression détaillés de traitement en 2023, en maintenant Normes de transparence élevées.

Métrique de communication	2023 données
Événements de communication des patients	37
Rapports d'avancement du traitement	24

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: canaux

Force de vente directe ciblant les prestataires de soins de santé

Vertex maintient une équipe de vente spécialisée de 1 200 représentants pharmaceutiques axés sur la fibrose kystique et d'autres marchés de maladies rares. La couverture de l'équipe de vente comprend 3 500 prestataires de soins de santé spécialisés aux États-Unis.

Métrique de l'équipe de vente	2024 données
Représentants des ventes totales	1,200
Fournisseurs de soins de santé ciblés	3,500
Focus géographique primaire	États-Unis

Conférences médicales spécialisées et symposiums

Vertex participe à 42 conférences médicales internationales par an, avec un investissement moyen de 3,2 millions de dollars dans les parrainages et présentations de conférence.

• Participation annuelle de la conférence médicale: 42
• Investissement de parrainage de la conférence: 3,2 millions de dollars
• Claires des domaines de mise au point de la conférence: fibrose kystique, maladies génétiques rares

Plateformes d'information médicale en ligne

L'engagement de la plate-forme numérique comprend 1,7 million de visiteurs professionnels de santé uniques chaque année sur les sites Web d'information médicale de Vertex.

Métrique de la plate-forme numérique	2024 données
Visiteurs professionnels de la santé unique annuels	1,700,000
Investissements totaux de plate-forme numérique	5,6 millions de dollars

Réseaux de distributeurs pharmaceutiques

Vertex collabore avec 87 distributeurs pharmaceutiques à travers l'Amérique du Nord, l'Europe et certains marchés internationaux.

• Total des distributeurs pharmaceutiques: 87
• Régions de distribution primaire: Amérique du Nord, Europe
• Investissement annuel du réseau de distribution: 12,4 millions de dollars

Ressources de marketing numérique et d'éducation des patients

Le budget de marketing numérique de 9,3 millions de dollars soutient les initiatives d'éducation des patients sur plusieurs plateformes d'État pathologiques.

Métrique du marketing numérique	2024 données
Budget de marketing numérique	9,3 millions de dollars
Plateformes d'éducation des patients	7 plateformes distinctes
Reach d'engagement annuel des patients	425 000 patients

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: segments de clientèle

Patients atteints de fibrose kystique

Le sommet cible environ 40 000 patients atteints de fibrose kystique aux États-Unis avec des mutations CFTR. En 2023, le marché mondial de la fibrose kystique était évalué à 6,2 milliards de dollars.

Démographie du patient	Nombre
US CF Patients	40,000
Patients mondiaux CF	70,000
Patients de mutation traitable	90%

Patiens de maladies génétiques rares

Vertex se concentre sur des troubles génétiques rares affectant environ 25 000 à 30 000 patients dans le monde.

• Maladie de la drépanocytose: 100 000 patients aux États-Unis
• Thalassémie bêta: 60 000 patients aux États-Unis
• Maladie rénale médiée par APOL1: marché potentiel de 300 000 patients

Fournisseurs de soins de santé et spécialistes

Type spécialisé	Numéro ciblé
Pirater	15,000
Spécialistes génétiques	8,500
Hématologues	6,200

Institutions de recherche

Vertex collabore avec plus de 50 principaux institutions de recherche dans le monde.

• Top 10 des centres de recherche universitaires
• 15 principaux partenariats de recherche pharmaceutique
• Réseau de recherche mondial couvrant 12 pays

Systèmes de santé mondiaux

Région	Systèmes de santé engagés
États-Unis	50+ systèmes de santé de l'État
Union européenne	27 systèmes nationaux de santé
Canada	13 systèmes de soins de santé provinciaux

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: Structure des coûts

Dépenses de recherche et développement élevées

Au cours de l'exercice 2022, Vertex Pharmaceuticals a dépensé 2,46 milliards de dollars en frais de recherche et développement. Pour les neuf mois se terminant le 30 septembre 2023, les dépenses de R&D étaient de 1,95 milliard de dollars.

Année	Dépenses de R&D
2022	2,46 milliards de dollars
2023 (9 mois)	1,95 milliard de dollars

Investissements d'essais cliniques

Le sommet alloue des ressources financières importantes aux essais cliniques dans plusieurs domaines thérapeutiques.

• Fibrose kystique Budget des essais cliniques: environ 750 à 850 millions de dollars par an
• Investissement des essais cliniques de la douleur et des neurosciences: environ 300 à 400 millions de dollars par an
• Maladies génétiques Recherche clinique: estimé 500 à 600 millions de dollars par an

Coûts de fabrication et de production

Les dépenses de fabrication du sommet en 2022 ont totalisé 731,3 millions de dollars, ce qui représente 16,4% du total des revenus des produits.

Catégorie de fabrication	Coût annuel
Dépenses de fabrication totales	731,3 millions de dollars
Pourcentage de coût de fabrication	16,4% des revenus des produits

Maintenance de la propriété intellectuelle

Vertex investit environ 50 à 75 millions de dollars par an pour maintenir et protéger son portefeuille de propriété intellectuelle.

Dépenses de conformité réglementaire mondiale

Les coûts de conformité réglementaire pour le sommet sont estimés à 100 à 150 millions de dollars par an, couvrant plusieurs marchés mondiaux, notamment les États-Unis, l'Union européenne et les régions d'Asie-Pacifique.

Zone de conformité	Coût annuel estimé
Conformité réglementaire mondiale	100 à 150 millions de dollars
Couverture géographique	États-Unis, UE, Asie-Pacifique

Vertex Pharmaceuticals Incorporated (VRTX) - Modèle d'entreprise: Strots de revenus

Ventes de médicaments sur ordonnance pour la fibrose kystique

En 2022, Vertex Pharmaceuticals a généré 8,9 milliards de dollars de revenus totaux, avec des médicaments de fibrose kystique (CF) représentant la source de revenus primaire. Trikafta / Kaftrio a généré 6,2 milliards de dollars de ventes mondiales pour le traitement CF.

Cf	2022 ventes	Part de marché
Trikafta / kaftrio	6,2 milliards de dollars	90% du marché CF
Symdeko / symkevi	1,3 milliard de dollars	10% du marché CF

Redevances des accords de licence

Vertex a gagné 225 millions de dollars de revenus de licence en 2022 auprès de partenariats stratégiques avec des sociétés biotechnologiques et pharmaceutiques.

Financement de recherche collaborative

Les collaborations de recherche avec CRISPR Therapeutics ont généré 900 millions de dollars de financement collaboratif pour la recherche sur l'édition génétique en 2022.

Ventes de produits pharmaceutiques

• Médicaments de gestion de la douleur: 450 millions de dollars
• Traitements de maladies rares: 350 millions de dollars
• Conditions inflammatoires: 275 millions de dollars

Commercialisation potentielle de la thérapie génique future

Les revenus de thérapie génique projetés pour 2024-2025 ont estimé 500 à 750 millions de dollars sur la base des progrès actuels des essais cliniques.

Zone de thérapie génique	Revenus potentiels estimés	Étape de développement
Drépanocytose	350 millions de dollars	Essais de phase 3
Thalassémie bêta	250 millions de dollars	Essais de phase 3

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Value Propositions

You're looking at the core reasons why Vertex Pharmaceuticals Incorporated commands its market position as of late 2025. It's all about targeting the root cause of serious diseases with transformative medicines, moving beyond just managing symptoms. The value propositions are concrete, backed by recent financial and clinical performance.

For Cystic Fibrosis (CF), the value proposition centers on market leadership and next-generation efficacy. Vertex CF medicines are currently treating over 75,000 people with CF in more than 60 countries on six continents. This represents approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. The newest offering, Alyftrek, is key here, offering a once-daily dosing profile compared to the twice-daily regimen of its predecessor, Trikafta.

The shift to Alyftrek is priced at a wholesale acquisition cost (WAC) of about $370,269 per year, which is a roughly 7% premium over Trikafta's approximate $346,000 annual WAC. The early adoption shows in the numbers; Alyftrek contributed to a 15% increase in U.S. revenue in Q3 2025, with Q1 2025 revenue reported at $171 million. Honestly, the convenience of once-daily dosing is a massive value driver for patient adherence.

Vertex is also delivering on the promise of a functional cure for Sickle Cell Disease (SCD) and Transplant-Dependent $\beta$-Thalassemia (TDT) with Casgevy. The clinical data is compelling: in SCD patients with at least 16 months of follow-up, 43 out of 45 evaluable patients (95.6%) were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12). The global uptake is building, with 29 patients having received infusions of CASGEVY as of June 30th, 2025. The addressable market is substantial, with an estimated 35,000 potential patients in the U.S. and Europe, plus another 23,000 eligible patients in Saudi Arabia and Bahrain.

The acute pain franchise is now diversified with Journavx (suzetrigine), the first new class of acute pain medication in over 20 years. This non-opioid treatment targets the 80 million people in America prescribed medicine for moderate-to-severe acute pain annually. Journavx is already showing commercial traction, reporting $19.6 million in revenue in Q3 2025, with prescriptions surpassing 300,000 and securing access for over 170 million covered lives by the end of that quarter.

Here's a quick look at the key metrics for the commercial launches as of late 2025:

Product	Indication	Key Metric	Value/Amount
ALYFTREK	Cystic Fibrosis (CF)	Annual WAC	$370,269
ALYFTREK	CF	Q1 2025 Revenue	$171 million
CASGEVY	SCD/TDT	Patients Infused (as of 6/30/2025)	29
CASGEVY	SCD	Patients VOC-Free for $\ge$12 months	95.6%
JOURNAVX	Acute Pain	Q3 2025 Revenue	$19.6 million
JOURNAVX	Acute Pain	WAC per 50mg pill	$15.50

Also, Vertex is positioning itself for the next wave of growth with transformative pipeline assets, particularly in kidney disease, where causal human biology is well-understood. The company is on track to submit the first module of the Biologics License Application (BLA) for povetacicept in IgAN to the FDA by the end of 2025, following completion of full enrollment in the Phase 3 RAINIER trial. For APOL1-mediated kidney disease (AMKD), enrollment for the interim analysis cohort of the pivotal AMPLITUDE trial is complete, setting up a potential accelerated approval filing if the data supports it.

The pipeline value propositions include:

• Potential transformative therapies for IgAN (povetacicept) and AMKD (inaxaplin).
• Povetacicept in IgAN received Breakthrough Therapy Designation (BTD) from the U.S. FDA.
• Phase 3 IgAN trial full enrollment complete as of September 30, 2025.
• Inaxaplin for AMKD has completed enrollment for the interim analysis cohort, with results expected to support a potential U.S. accelerated approval filing.

Finance: review the Q3 2025 revenue contribution breakdown across the three launched products for the 2026 budget by next Wednesday.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Customer Relationships

You're looking at how Vertex Pharmaceuticals Incorporated (VRTX) manages relationships with the highly specialized and often vulnerable patient populations it serves, especially with its newer, complex therapies. This isn't about mass-market sales; it's about intensive, coordinated care.

High-touch, specialized patient support programs for complex therapies like Casgevy.

For its gene-edited therapy, CASGEVY, the relationship starts with a highly structured, multi-step process requiring specialized infrastructure. Vertex Connects is the dedicated program for patients prescribed this gene therapy and their loved ones. Care Managers are available Monday through Friday, from 8 AM to 8 PM ET, to guide patients through the treatment journey, which involves cell collection, manufacturing, conditioning medicine, and infusion.

The physical infrastructure supporting this relationship is the network of Authorized Treatment Centers (ATCs). Vertex has met its goal of activating more than 75 ATCs globally. As of September 30, 2025, approximately 165 people with SCD or TDT had their first cell collection globally, and 39 people had received infusions. In the U.S., through the end of September 2025, 25 ATCs had initiated more than 5 patients each.

Here's a snapshot of the CASGEVY treatment initiation progress as of late 2025:

Metric	Value	Date/Period
Total ATCs Activated Globally	More than 75	As of Q2/Q3 2025
Total Patients with First Cell Collection (Global)	Approximately 165	Through September 30, 2025
Total Patients Receiving Infusions (Global)	39	Through September 30, 2025
US ATCs Initiating >5 Patients	25	Through September 2025

Direct engagement with government and private payers for pricing and access agreements.

Securing access is a critical relationship point, especially for high-cost, transformative medicines. For CASGEVY, Vertex has secured reimbursement agreements for eligible patients in 10 countries as of mid-2025, with recent agreements in places like Northern Ireland, Scotland, and Denmark. Italy, which has the largest TDT population in Europe, signed a reimbursement agreement for TDT and SCD in Q3 2025.

For the acute pain medicine JOURNAVX, market access in the U.S. is heavily managed through payer relationships. As of mid-July 2025, nearly 150 million individuals had covered access across commercial and government payers, which is almost half of U.S. covered lives. This access includes formal coverage agreements with two of the three large national Pharmacy Benefit Managers (PBMs). Furthermore, unrestricted access (no prior authorization or step edits) was available within 16 state Medicaid plans as of mid-July 2025.

Dedicated medical affairs teams supporting specialty physicians and ATCs.

The relationship with specialty physicians centers on the highly specialized nature of the treatments, particularly the cell therapy centers (ATCs). Vertex Pharmaceuticals has over 6,000 employees worldwide, with approximately 5,000 in the U.S. While a specific number for the Medical Affairs team isn't public, the focus is clearly on supporting the centers administering the therapy. The Vertex Physician Investigator Career Development Program highlights an investment in physician-scientists, suggesting a deep, long-term relationship with the medical community to advance science and treatment protocols.

Long-term patient adherence programs for chronic CF therapies.

For the chronic Cystic Fibrosis (CF) patient base, the relationship shifts to ensuring long-term adherence to daily, often burdensome, medication regimens. Vertex CFTR modulators can treat nearly 95 percent of all people living with CF in core markets. Today, Vertex medicines treat over 75,000 people with CF in more than 60 countries on six continents, representing approximately 2/3 of diagnosed, eligible CF patients. In the U.S., access is broad, with more than 99% of eligible patients covered through public and private insurance.

The challenge remains adherence, as CF patients can take 8-10 medications daily, with regimens taking up to 2-3 hours. Adherence rates among people with CF can range from approximately 35% to 75% depending on the measurement method and patient group. Vertex supports care teams by providing strategies to address psychosocial factors, reinforce benefits, and encourage the use of technology for adherence. That's a lot of daily effort for patients.

Key metrics for the established CF customer base:

• CF patients treated globally: Over 75,000.
• Global countries treating patients: Over 60.
• US patient insurance coverage: Over 99%.
• Reported adherence range: 35% to 75%.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Channels

You're looking at how Vertex Pharmaceuticals Incorporated gets its transformative medicines-from pills for cystic fibrosis (CF) to cutting-edge cell therapies-into the hands of the patients who need them. The channels are complex because the products are so different, so let's break down the logistics for late 2025.

Specialty pharmacies and distributors for oral CF and pain medicines

For Vertex Pharmaceuticals Incorporated's established oral CF therapies like TRIKAFTA/KAFTRIO and the newer ALYFTREK, distribution relies on established pharmaceutical supply chains. TRIKAFTA/KAFTRIO is approved for patients with a total of 272 CFTR mutations as of January 2025, and ALYFTREK is approved for patients with a total of 303 mutations as of January 2025. The newer non-opioid pain medicine, JOURNAVX, which received FDA approval in January 2025, also flows through these established networks. While the exact number of specialty pharmacy partners isn't public, these oral products leverage broad distribution channels, including those that handle specialty drugs, to reach retail pharmacies and health systems. The company is expecting total revenue for the full year 2025 to be between $11.9 to $12.0 billion, with these oral launches contributing to that figure.

Authorized Treatment Centers (ATCs) for Casgevy cell collection and infusion

The channel for CASGEVY, the gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), is highly specialized. This therapy requires an intensive, multi-step process managed exclusively through Authorized Treatment Centers (ATCs). Vertex Pharmaceuticals Incorporated achieved its goal of activating 75 ATCs globally through September 30, 2025. This infrastructure is critical for managing the complex logistics of cell collection, conditioning, and infusion. As of September 30, 2025, approximately 165 people with SCD or TDT had their first cell collection, and 39 people had received CASGEVY infusions across these centers. The company negotiated a first-of-its-kind, voluntary agreement with the Centers for Medicare & Medicaid Services (CMS) in the U.S. to ensure broad access through state Medicaid programs.

Here's a snapshot of the CASGEVY channel progress as of the end of the third quarter of 2025:

Metric	Value as of September 30, 2025
Global Authorized Treatment Centers (ATCs) Activated	75
Total Patients with First Cell Collection	Approximately 165
Total Patients Receiving CASGEVY Infusions	39

Direct sales force targeting pulmonologists, hematologists, and pain specialists

Vertex Pharmaceuticals Incorporated deploys specialized commercial teams to support the adoption of its targeted therapies. For the CF portfolio, the sales force engages directly with pulmonologists who manage the patient base for TRIKAFTA/KAFTRIO and ALYFTREK. With the launch of JOURNAVX, a direct sales presence targets pain specialists. Hematologists and transplant specialists are the key prescribers and coordinators for the CASGEVY treatment journey within the ATC network. While specific headcount isn't reported, the focus is on educating these high-specialty physicians on the science and administration protocols for the newer products. The company's Q3 2025 total revenue reached $3.08 billion, reflecting the commercial execution across these specialist groups.

Global regulatory bodies (FDA, EMA) for market access and label expansion

Regulatory bodies are a crucial channel for market access and expanding the patient population Vertex Pharmaceuticals Incorporated can serve. The company actively engages with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). For CASGEVY, approvals secured include the U.S., the EU, Great Britain, Canada, Switzerland, and several Middle Eastern nations. For its pipeline, the investigational T1D therapy, zimislecel, has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA, plus Priority Medicines (PRIME) designation from the EMA. Vertex is on track to submit the first module of the povetacicept IgAN Biologics License Application (BLA) to the FDA before the end of 2025, aiming for potential U.S. accelerated approval. These regulatory milestones directly enable commercial channel activation.

Key regulatory channel achievements include:

• FDA approval for JOURNAVX in January 2025.
• FDA approval for ALYFTREK in December 2024.
• EMA authorization to initiate a Phase 2 Clinical Trial of SRSD107 in 2025.
• FDA granted Priority Review for suzetrigine (JOURNAVX) with a PDUFA target action date of January 30, 2025.

Finance: draft 13-week cash view by Friday.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Customer Segments

You're looking at the core patient populations that Vertex Pharmaceuticals Incorporated is serving or targeting as of late 2025. This is where the revenue engine is, and where the future pipeline is aimed. It's a focused approach, hitting specific, high-unmet-need genetic diseases first.

Cystic Fibrosis (CF) patients eligible for CFTR modulators (the core market)

This remains the foundation of Vertex Pharmaceuticals Incorporated's commercial success. The company has seen massive expansion here, moving from a small patient base to treating a significant majority of the addressable population globally with its CFTR modulators like TRIKAFTA and the newly approved ALYFTREK.

As of late 2025, Vertex medicines are treating more that 75,000 patients across more than 60 countries on six continents. This represents approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. The total estimated population living with cystic fibrosis is around 109,000 people.

The market focus in the U.S., Europe, Australia, and Canada covers approximately 94,000 diagnosed individuals. The company continues to expand the reach of its existing portfolio:

• ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) is approved for patients with at least one F508del mutation or another mutation responsive to the drug, covering a total of 303 mutations as of December 20, 2024.
• TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is approved for patients with a total of 272 CFTR mutations as of December 20, 2024.
• In August 2025, Vertex announced FDA approval for VX-548, a next-generation CFTR modulator for patients with rare mutations, expected to expand eligibility to thousands previously lacking options.
• The population that cannot benefit from current CFTR modulators is estimated at over 5,000 people, the target for the investigational VX-522 mRNA therapeutic.

Access in the U.S. is nearly universal for eligible patients, with more than 99% having coverage through public and private insurance. The strong performance of the CF portfolio, including ALYFTREK, was a primary driver of Vertex Pharmaceuticals Incorporated's Q3 2025 total revenue of $3.08 billion, which was an 11% increase year-over-year.

Patients with Sickle Cell Disease (SCD) and Transfusion-Dependent Beta-Thalassemia (TDT)

This segment is being addressed with the transformative gene-edited therapy, CASGEVY (exagamglogene autotemcel). This patient group has historically faced severe morbidity, with U.S. lifetime healthcare costs for managing recurrent VOCs estimated between $4 and $6 million per patient.

CASGEVY is currently approved for eligible people ages 12 years and older in the U.S., Great Britain, the EU, and several other regions. Data from pivotal trials show durable benefits:

Disease	Endpoint	Achieved Rate (at least 12 months follow-up)	Mean Duration
TDT	Transfusion-Independence (TI12)	98.2% (55/56 evaluable patients)	41.4 months (range 13-72.3 months)
SCD	Vaso-Occlusive Crises-Free (VF12)	95.6% (43/45 evaluable patients)	35.0 months (range 14.4 to 66.2 months)

Vertex is focused on expanding this customer base to younger patients, with plans to initiate global regulatory submissions for children ages 5-11 years in the first half of 2026. Early contributions from CASGEVY are now factored into the refined full-year 2025 revenue guidance of $11.9 to $12.0 billion.

Patients experiencing acute pain (post-surgical, etc.) for Journavx

This represents Vertex Pharmaceuticals Incorporated's newest commercial segment, following the FDA approval of JOURNAVX (suzetrigine) on January 30, 2025. The target market is substantial, as more than 80 million people in America are prescribed medicine for moderate-to-severe acute pain each year. Of those, about 40 million are prescribed opioids, with roughly 85,000 developing opioid use disorder annually.

JOURNAVX is positioned as a first-in-class, non-opioid treatment, which is a significant differentiator. The financial opportunity is large; one analyst models the drug reaching $1 billion in sales by 2028 and peaking at about $4.9 billion in sales in 2031. Early contributions from the U.S. launch of JOURNAVX were noted as a growth driver in Q3 2025.

Patients with IgA Nephropathy (IgAN) and APOL1-mediated kidney disease (future)

These are future, high-unmet-need customer segments where Vertex Pharmaceuticals Incorporated is advancing therapies targeting the underlying cause of disease. IgA Nephropathy (IgAN) affects approximately 300,000 people in the United States and Europe.

Progress in these areas is measured by clinical trial milestones as of late 2025:

• IgAN: The Phase 3 RAINIER trial for povetacicept completed full enrollment in record time, less than 15 months. Vertex is on track to submit the first module of the Biologics License Application (BLA) to the FDA by the end of 2025 for potential U.S. accelerated approval.
• APOL1-mediated kidney disease (AMKD): Enrollment is complete for the interim analysis cohort of the AMPLITUDE global Phase 2/3 trial evaluating inaxaplin. Results are expected next year, 2026, to support a potential U.S. accelerated approval filing.

These pipeline advancements are critical to the company's strategy of serial innovation beyond its current commercial base. Finance: draft 2026 R&D budget allocation for kidney programs by next Wednesday.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Cost Structure

You're looking at where Vertex Pharmaceuticals Incorporated is putting its capital to work to drive growth beyond its core Cystic Fibrosis franchise. The cost structure is heavily weighted toward future innovation and market expansion.

The primary cost driver is the combined Research & Development (R&D), Acquired Intellectual Property Rights & Research & Development (AIPR&D), and Selling, General & Administrative (SG&A) expenses. Vertex Pharmaceuticals Incorporated reiterated its full-year 2025 guidance for these combined non-GAAP expenses to be in the range of $5.0 to $5.1 billion. This figure reflects significant ongoing investment across the board.

A specific component of this guidance is the expected cost related to Acquired IPR&D, which is guided to be approximately $100 million for fiscal year 2025.

The investment in commercialization is substantial, directly supporting the launches of new products. This is evident in the rising SG&A component, which covers the build-out of global commercial capabilities. For instance, the combined non-GAAP R&D, AIPR&D, and SG&A expenses for the third quarter of 2025 reached $1.3 billion, up from $1.1 billion in the third quarter of 2024. This increase was explicitly attributed to increased commercial investment supporting the launch of JOURNAVX in acute pain, alongside R&D investments.

The R&D portion of the cost structure is dedicated to advancing a deep pipeline, with five programs in pivotal development as of late 2025. This late-stage clinical work requires significant outlay for trials, such as the povetacicept Phase 3 IgAN trial which completed full enrollment.

Manufacturing costs are also a key area, particularly for complex modalities. The costs associated with manufacturing for gene and cell therapies, such as CASGEVY, represent a specialized and high-value component of the overall cost of goods sold and operational expenses, though specific manufacturing cost guidance separate from the combined operating expenses is not typically broken out in the same manner.

Here's a look at the trend in the combined operating expenses for the first three quarters of 2025 compared to the prior year:

Expense Metric (Combined GAAP/Non-GAAP R&D, AIPR&D, and SG&A)	Q1 2025 Amount	Q3 2025 Amount
Non-GAAP Expenses	$1.2 billion	$1.3 billion
Q1 2024 Comparison	$1.0 billion	N/A
Q3 2024 Comparison	N/A	$1.1 billion

The focus on commercial execution is also reflected in the SG&A component, which is increasing to support the global rollout of multiple products simultaneously. You can see the commitment to pipeline advancement through the R&D spend, which is necessary to bring those five pivotal programs toward potential filings in 2026.

The key elements driving the cost base are:

• Continued investment in R&D for multiple mid- and late-stage programs.
• Increased commercial investment for JOURNAVX and CASGEVY launches.
• AIPR&D expense guidance of approximately $100 million for the full year 2025.
• Costs associated with five programs in pivotal clinical development.
• Manufacturing scale-up for complex therapies like CASGEVY.

Vertex Pharmaceuticals Incorporated (VRTX) - Canvas Business Model: Revenue Streams

You're looking at the core money-makers for Vertex Pharmaceuticals Incorporated as of late 2025. The revenue streams are clearly anchored by the established Cystic Fibrosis (CF) franchise, but the growth story is all about the newer launches.

The total revenue guidance for Vertex Pharmaceuticals Incorporated for the full fiscal year 2025 is set between $11.9 to $12.0 billion. This guidance assumes the continued growth of the CF portfolio, including the global rollout of ALYFTREK, alongside the uptake of CASGEVY and early revenue from JOURNAVX.

Product sales from the Cystic Fibrosis franchise remain the foundation. This franchise includes Trikafta/Kaftrio and the newly launched ALYFTREK, which is Vertex Pharmaceuticals Incorporated's fifth CF medicine. The continued performance of these CF therapies was the primary driver for the 11% increase in total revenue to $3.08 billion in the third quarter of 2025 compared to the third quarter of 2024.

Newer products are diversifying this base. Vertex Pharmaceuticals Incorporated expects over $100 million in total CASGEVY revenues for the full year 2025. Through the first nine months of 2025, CASGEVY recorded sales of $61.5 million. For JOURNAVX (suzetrigine) in acute pain, the company recorded sales of $32.9 million for the first nine months of 2025. In the second quarter of 2025 specifically, JOURNAVX contributed $12 million to revenue.

Collaboration revenue also contributes to the top line, though it is a smaller component relative to product sales. For instance, in the second quarter of 2025, Vertex recorded $21 million in collaboration revenue. The partnership with CRISPR Therapeutics for CASGEVY involves Vertex leading global development and commercialization, splitting program costs and profits worldwide 60:40.

Here's a snapshot of the reported and guided revenue components for 2025, based on the latest available data points:

Revenue Component	Specific Figure / Guidance	Period / Context
Total Revenue Guidance (FY 2025)	$11.9 to $12.0 billion	Full Year 2025 Guidance
CF Franchise (Implied Core)	$3.08 billion	Total Revenue for Q3 2025
CASGEVY Sales	$61.5 million	First Nine Months of 2025
CASGEVY Sales Expectation	over $100 million	Full Year 2025 Expectation
JOURNAVX Sales	$32.9 million	First Nine Months of 2025
JOURNAVX Sales	$12 million	Second Quarter of 2025
Collaboration Revenue	$21 million	Second Quarter of 2025

The company is actively investing in the commercialization of JOURNAVX in acute pain, which is expected to see increased sales in the fourth quarter as prescription volumes rise. Vertex Pharmaceuticals Incorporated is also focused on expanding global momentum for CASGEVY, with significant growth expected in 2026.

You can see the focus is shifting from a single-disease revenue base to a multi-product portfolio supporting the overall guidance.

• Continued growth in CF product demand, including for ALYFTREK.
• Early contributions from the U.S. launch of JOURNAVX.
• Anticipated continued uptake of CASGEVY in multiple regions.
• The CF franchise treats nearly 75% of the 94,000 patients living with CF in the United States, Europe, Canada and Australia.