Vertex Pharmaceuticals Incorporated (VRTX): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el mundo de la biotecnología de vanguardia, Vertex Pharmaceuticals se erige como un faro de esperanza, transformando desafíos genéticos complejos en soluciones médicas innovadoras. Con un enfoque centrado en el láser en enfermedades raras como la fibrosis quística, esta empresa innovadora ha revolucionado la medicina de precisión a través de tecnologías avanzadas de edición de genes y colaboraciones estratégicas. Su modelo de negocio representa un plano sofisticado de innovación científica, atención centrada en el paciente y estrategias de atención médica transformadora que prometen redefinir cómo entendemos y tratamos los trastornos genéticos.

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: asociaciones clave

Colaboración estratégica con CRISPR Therapeutics

En enero de 2022, Vertex y CRISPR Therapeutics extendieron su acuerdo de colaboración para las terapias de edición de genes. La asociación se centra en el desarrollo de CTX001 para la enfermedad de las células falciformes y la beta-talasemia. A partir de 2023, la colaboración tiene un valor total de $ 900 millones en posibles pagos de hitos.

Detalles de la asociación	Valor financiero
Duración del acuerdo de colaboración	Extendido hasta 2022-2024
Pagos potenciales de hitos	$ 900 millones
Enfoque principal	Terapias de edición de genes para enfermedades genéticas

Asociaciones de investigación con instituciones académicas

Vertex mantiene colaboraciones de investigación estratégica con múltiples centros académicos:

• Hospital de Niños de Boston
• Hospital General de Massachusetts
• Universidad de Stanford
• Universidad de California, San Francisco

Institución	Enfoque de investigación	Año de colaboración
Hospital de Niños de Boston	Investigación de fibrosis quística	2018
Universidad de Stanford	Terapias con células madre	2020

Acuerdos de licencia

Vertex ha establecido acuerdos de licencia con múltiples compañías de biotecnología:

• Wuxi Biologics (China): Asociación de fabricación
• Moderna: colaboraciones terapéuticas de ARNm
• Seagen: desarrollo oncológico de medicamentos

Pareja	Tipo de acuerdo	Valor estimado
Wuxi Biologics	Licencia de fabricación	$ 250 millones
Moderna	colaboración terapéutica de ARNm	$ 100 millones por adelantado

Colaboraciones organizacionales de atención médica global

Vertex se asocia con organizaciones internacionales de salud para el desarrollo de medicamentos y ensayos clínicos en múltiples áreas terapéuticas.

• Organización Mundial de la Salud (OMS)
• Agencia Europea de Medicamentos (EMA)
• Institutos Nacionales de Salud (NIH)

Organización	Enfoque de colaboración	Año de compromiso
OMS	Iniciativas de salud globales	2021
NIH	Investigación de enfermedades raras	2019

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: actividades clave

Investigación y desarrollo de tratamientos innovadores de fibrosis quística

Vertex invirtió $ 2.1 mil millones en gastos de I + D en 2022, con un enfoque significativo en las terapias de fibrosis quística. La cartera del modulador CFTR de la compañía generó $ 8.4 mil millones en ingresos en 2022.

I + D Métrica	Valor 2022
Gastos totales de I + D	$ 2.1 mil millones
Ingresos de la cartera de fibrosis quística	$ 8.4 mil millones

Ensayos clínicos para terapias de enfermedad genética raras

A partir de 2023, Vertex mantuvo 15 ensayos clínicos activos En múltiples áreas terapéuticas, incluidas enfermedades genéticas raras.

• Ensayos clínicos en curso en enfermedad de células falciformes
• Programas de investigación de diabetes tipo 1
• Estudios de deficiencia de antitripsina alfa-1

Descubrimiento y desarrollo farmacéutico de fármacos

Métrica de descubrimiento de drogas	2022-2023 datos
Nuevas solicitudes de drogas presentadas	3 aplicaciones
Candidatos de tuberías	20+ terapias potenciales

Fabricación de terapias genéticas y celulares especializadas

Vertex opera instalaciones de fabricación en Massachusetts y California con una capacidad de producción total de aproximadamente 500,000 unidades de tratamiento anualmente.

Inversión continua en investigación de biotecnología avanzada

Investigación de desglose de inversión para 2022:

Área de investigación	Inversión
Fibrosis quística	$ 1.2 mil millones
Enfermedades genéticas	$ 600 millones
Dolor y trastornos neurológicos	$ 300 millones

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: recursos clave

Cartera de propiedades intelectuales

A partir de 2024, Vertex Pharmaceuticals se mantiene Más de 1.200 patentes activas relacionado con la medicina genética y los tratamientos de fibrosis quística.

Categoría de patente	Número de patentes	Valor estimado
Fibrosis quística	450	$ 3.2 mil millones
Edición de genes	350	$ 2.7 mil millones
Terapias celulares	250	$ 1.9 mil millones

Investigaciones y instalaciones de desarrollo

Vértice opera 4 centros de investigación primarios ubicado en:

• Boston, Massachusetts (sede)
• San Diego, California
• Londres, Reino Unido
• Cambridge, Massachusetts

Talento científico y médico

A partir de 2024, Vertex emplea 3.650 profesionales de investigación y desarrollo.

Categoría de empleado	Número de empleados
Investigadores de doctorado	1,200
Médico	350
Investigadores clínicos	850

Tecnologías patentadas

Vertex se ha desarrollado 3 plataformas primarias de edición de genes:

• Sistema de modificación de genes basado en CRISPR
• Tecnología de reprogramación celular avanzada
• Plataforma de intervención genética de precisión

Recursos financieros

Métricas financieras a partir de 2024:

Métrica financiera	Cantidad
Presupuesto de investigación total	$ 1.8 mil millones
Efectivo e inversiones	$ 6.3 mil millones
Gastos anuales de I + D	$ 2.1 mil millones

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: propuestas de valor

Tratamientos innovadores para los trastornos genéticos

Vertex Pharmaceuticals se centra en el desarrollo de terapias dirigidas para enfermedades genéticas, con un énfasis primario en los tratamientos de fibrosis quística (CF).

Producto	Condición objetivo	Ingresos anuales (2023)
Trikafta/Kaftrio	Fibrosis quística	$ 8.4 mil millones
Symdeko/Orkambi	Fibrosis quística	$ 1.2 mil millones

Terapias dirigidas para pacientes con fibrosis quística

Vértice ha desarrollado una cartera integral de terapias CF que abordan múltiples mutaciones genéticas.

• Cubre aproximadamente el 90% de las mutaciones del paciente con FQ
• Opciones de tratamiento para pacientes mayores de 2 años
• Mejora mediana de la función pulmonar de 10.4 puntos porcentuales

Soluciones innovadoras de medicina de precisión

Área de investigación	Inversión (2023)	Número de pruebas en curso
Terapias genéticas	$ 2.1 mil millones	37 ensayos clínicos
Manejo del dolor	$ 380 millones	12 ensayos clínicos

Mejora de la calidad de vida del paciente

Las terapias de Vértice demuestran resultados clínicos significativos:

• Reducción de exacerbaciones pulmonares en un 63%
• Tasas de supervivencia del paciente mejoradas
• Función respiratoria mejorada

Enfoques científicos de vanguardia

Dominio de la investigación	Áreas de enfoque clave	Cartera de patentes
Medicina genética	Tecnología CRISPR	87 patentes activas
Enfermedades raras	Intervenciones genéticas de precisión	52 aplicaciones pendientes

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: relaciones con los clientes

Compromiso directo con grupos de apoyo para pacientes

Vertex Pharmaceuticals mantiene asociaciones activas con 87 organizaciones de apoyo a los pacientes con fibrosis quística a nivel mundial a partir de 2023. La compañía invirtió $ 3.2 millones en iniciativas de colaboración del grupo de apoyo de pacientes directos.

Métrica del grupo de apoyo al paciente	2023 datos
Número de organizaciones globales de pacientes	87
Inversión en colaboración grupal de apoyo	$ 3.2 millones

Servicios de consulta médica personalizada

Vertex ofrece servicios de consulta especializados a través de una red dedicada de 214 especialistas clínicos centrados en el manejo de enfermedades raras.

• Especialistas de consulta: 214
• Duración de consulta promedio: 45 minutos
• Tasa de satisfacción del paciente: 94%

Plataformas de salud digital para la gestión del paciente

La plataforma de salud digital de la compañía atiende a 12.500 usuarios activos con capacidades de monitoreo de tratamiento en tiempo real. La inversión en desarrollo de la plataforma alcanzó los $ 7.5 millones en 2023.

Métrica de plataforma digital	2023 datos
Usuarios de plataforma activa	12,500
Inversión de desarrollo de plataforma	$ 7.5 millones

Programas integrales de asistencia al paciente

Vertex opera programas de asistencia al paciente que cubren el 96% de los costos de medicamentos recetados para pacientes elegibles. El gasto total del programa en 2023 fue de $ 42.6 millones.

• Cobertura de costos de medicación: 96%
• Gasto total de asistencia al paciente: $ 42.6 millones
• Número de pacientes apoyados: 8,700

Comunicación transparente sobre desarrollos de tratamiento

La Compañía realizó 37 eventos de comunicación del paciente y publicó 24 informes detallados de progreso del tratamiento en 2023, manteniendo Estándares de alta transparencia.

Métrico de comunicación	2023 datos
Eventos de comunicación del paciente	37
Informes de progreso del tratamiento	24

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: canales

Fuerza de ventas directa dirigida a proveedores de atención médica

Vertex mantiene un equipo de ventas especializado de 1.200 representantes farmacéuticos centrados en la fibrosis quística y otros mercados de enfermedades raras. La cobertura del equipo de ventas incluye 3.500 proveedores de atención médica especializados en los Estados Unidos.

Métrica del equipo de ventas	2024 datos
Representantes de ventas totales	1,200
Proveedores de atención médica dirigidos	3,500
Enfoque geográfico primario	Estados Unidos

Conferencias y simposios médicos especializados

Vertex participa en 42 conferencias médicas internacionales anualmente, con una inversión promedio de $ 3.2 millones en patrocinios y presentaciones de conferencias.

• Participación anual de la conferencia médica: 42
• Inversión de patrocinio de la conferencia: $ 3.2 millones
• Áreas clave de enfoque de la conferencia: fibrosis quística, enfermedades genéticas raras

Plataformas de información médica en línea

La participación de la plataforma digital incluye 1,7 millones de visitantes profesionales de atención médica únicos anualmente en los sitios web de información médica de Vértice.

Métrica de plataforma digital	2024 datos
Visitantes anuales de profesionales de la salud únicos	1,700,000
Inversiones totales de plataforma digital	$ 5.6 millones

Redes de distribuidores farmacéuticos

Vertex colabora con 87 distribuidores farmacéuticos en América del Norte, Europa y mercados internacionales seleccionados.

• Distribuidores farmacéuticos totales: 87
• Regiones de distribución primaria: América del Norte, Europa
• Inversión de red de distribución anual: $ 12.4 millones

Marketing digital y recursos de educación del paciente

El presupuesto de marketing digital de $ 9.3 millones apoya las iniciativas de educación del paciente en múltiples plataformas estatales de enfermedades.

Métrica de marketing digital	2024 datos
Presupuesto de marketing digital	$ 9.3 millones
Plataformas de educación para pacientes	7 plataformas distintas
Alcance anual de compromiso del paciente	425,000 pacientes

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: segmentos de clientes

Pacientes con fibrosis quística

Vértice se dirige a aproximadamente 40,000 pacientes con fibrosis quística en los Estados Unidos con mutaciones CFTR. A partir de 2023, el mercado global de fibrosis quística se valoró en $ 6.2 mil millones.

Demografía del paciente	Número
Pacientes de FQ US	40,000
Pacientes globales de FQ	70,000
Pacientes de mutación tratable	90%

Pacientes de enfermedad genética rara

Vertex se centra en trastornos genéticos raros que afectan a aproximadamente 25,000-30,000 pacientes a nivel mundial.

• Enfermedad de células falciformes: 100,000 pacientes en EE. UU.
• Talasemia beta: 60,000 pacientes en EE. UU.
• Enfermedad renal mediada por APOL1: mercado potencial de 300,000 pacientes

Proveedores de atención médica y especialistas

Tipo especialista	Número dirigido
Pulmonólogos	15,000
Especialistas genéticos	8,500
Hematólogos	6,200

Instituciones de investigación

Vertex colabora con más de 50 instituciones de investigación líderes en todo el mundo.

• Top 10 centros de investigación académicos
• 15 Asociaciones importantes de investigación farmacéutica
• Red de investigación global que abarca 12 países

Sistemas de atención médica global

Región	Sistemas de atención médica comprometidos
Estados Unidos	50+ sistemas estatales de atención médica
unión Europea	27 sistemas nacionales de atención médica
Canadá	13 sistemas de salud provinciales

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: Estructura de costos

Altos gastos de investigación y desarrollo

En el año fiscal 2022, Vertex Pharmaceuticals gastó $ 2.46 mil millones en gastos de investigación y desarrollo. Para los nueve meses que terminaron el 30 de septiembre de 2023, los gastos de I + D fueron de $ 1.95 mil millones.

Año	Gastos de I + D
2022	$ 2.46 mil millones
2023 (9 meses)	$ 1.95 mil millones

Inversiones de ensayos clínicos

Vertex asigna recursos financieros significativos a ensayos clínicos en múltiples áreas terapéuticas.

• Presupuesto de ensayos clínicos de fibrosis quística: aproximadamente $ 750-850 millones anuales
• Inversión de ensayos clínicos de dolor y neurociencia: alrededor de $ 300-400 millones por año
• Investigación clínica de enfermedades genéticas: estimado de $ 500-600 millones anualmente

Costos de fabricación y producción

Los gastos de fabricación para el vértice en 2022 totalizaron $ 731.3 millones, lo que representa el 16.4% de los ingresos totales del producto.

Categoría de fabricación	Costo anual
Gastos totales de fabricación	$ 731.3 millones
Porcentaje de costos de fabricación	16.4% de los ingresos del producto

Mantenimiento de la propiedad intelectual

Vertex invierte aproximadamente $ 50-75 millones anuales en el mantenimiento y la protección de su cartera de propiedades intelectuales.

Gastos de cumplimiento regulatorio global

Los costos de cumplimiento regulatorio para el vértice se estiman en $ 100-150 millones por año, que cubren múltiples mercados globales, incluidas las regiones de los Estados Unidos, la Unión Europea y el Pacífico de Asia.

Área de cumplimiento	Costo estimado anual
Cumplimiento regulatorio global	$ 100-150 millones
Cobertura geográfica	EE. UU., UE, Asia-Pacífico

Vertex Pharmaceuticals Incorporated (VRTX) - Modelo de negocio: flujos de ingresos

Venta de medicamentos recetados para fibrosis quística

En 2022, Vertex Pharmaceuticals generó $ 8.9 mil millones en ingresos totales, con fármacos de fibrosis quística (CF) que representan la fuente de ingresos primario. Trikafta/Kaftrio generó $ 6.2 mil millones en ventas globales para tratamiento con FQ.

Drogadicto	2022 Ventas	Cuota de mercado
Trikafta/Kaftrio	$ 6.2 mil millones	90% del mercado de CF
Symdeko/Symkevi	$ 1.3 mil millones	10% del mercado de CF

Regalías de acuerdos de licencia

Vertex ganó $ 225 millones en ingresos por licencias en 2022 de asociaciones estratégicas con biotecnología y compañías farmacéuticas.

Financiación de la investigación colaborativa

Las colaboraciones de investigación con Terapéutica CRISPR generaron $ 900 millones en fondos colaborativos para la investigación de edición de genes en 2022.

Venta de productos farmacéuticos

• Medicamentos de manejo del dolor: $ 450 millones
• Tratamientos de enfermedades raras: $ 350 millones
• Condiciones inflamatorias: $ 275 millones

Comercialización potencial de terapia génica futura

Ingresos de terapia génica proyectados para 2024-2025 estimados en $ 500- $ 750 millones según el progreso actual del ensayo clínico.

Área de terapia génica	Ingresos potenciales estimados	Etapa de desarrollo
Anemia drepanocítica	$ 350 millones	Pruebas de fase 3
Talasemia beta	$ 250 millones	Pruebas de fase 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.