Vertex Pharmaceuticals Incorporated (VRTX): Business Model Canvas

In der hochmodernen Welt der Biotechnologie gilt Vertex Pharmaceuticals als Hoffnungsträger, der komplexe genetische Herausforderungen in bahnbrechende medizinische Lösungen umwandelt. Mit einem laserfokussierten Ansatz für seltene Krankheiten wie Mukoviszidose hat dieses innovative Unternehmen die Präzisionsmedizin durch fortschrittliche Gen-Editing-Technologien und strategische Kooperationen revolutioniert. Ihr Geschäftsmodell stellt einen ausgefeilten Entwurf wissenschaftlicher Innovation, patientenzentrierter Versorgung und transformativer Gesundheitsstrategien dar, die versprechen, die Art und Weise, wie wir genetische Störungen verstehen und behandeln, neu zu definieren.

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Wichtige Partnerschaften

Strategische Zusammenarbeit mit CRISPR Therapeutics

Im Januar 2022 verlängerten Vertex und CRISPR Therapeutics ihre Kooperationsvereinbarung für Gen-Editing-Therapien. Die Partnerschaft konzentriert sich auf die Entwicklung von CTX001 zur Behandlung von Sichelzellanämie und Beta-Thalassämie. Ab 2023 hat die Zusammenarbeit einen Gesamtwert von 900 Millionen US-Dollar an potenziellen Meilensteinzahlungen.

Einzelheiten zur Partnerschaft	Finanzieller Wert
Dauer der Kooperationsvereinbarung	Verlängert bis 2022–2024
Mögliche Meilensteinzahlungen	900 Millionen Dollar
Hauptfokus	Geneditierende Therapien für genetische Krankheiten

Forschungskooperationen mit akademischen Institutionen

Vertex unterhält strategische Forschungskooperationen mit mehreren akademischen Zentren:

• Boston Kinderkrankenhaus
• Massachusetts General Hospital
• Stanford-Universität
• Universität von Kalifornien, San Francisco

Institution	Forschungsschwerpunkt	Jahr der Zusammenarbeit
Boston Kinderkrankenhaus	Mukoviszidose-Forschung	2018
Stanford-Universität	Stammzelltherapien	2020

Lizenzvereinbarungen

Vertex hat Lizenzvereinbarungen mit mehreren Biotechnologieunternehmen abgeschlossen:

• WuXi Biologics (China): Produktionspartnerschaft
• Moderna: mRNA-therapeutische Kooperationen
• Seagen: Entwicklung von Onkologiemedikamenten

Partner	Vereinbarungstyp	Geschätzter Wert
WuXi Biologics	Herstellungslizenz	250 Millionen Dollar
Moderna	mRNA-therapeutische Zusammenarbeit	100 Millionen Dollar im Voraus

Globale organisatorische Zusammenarbeit im Gesundheitswesen

Vertex arbeitet mit internationalen Gesundheitsorganisationen für die Arzneimittelentwicklung und klinische Studien in verschiedenen Therapiebereichen zusammen.

• Weltgesundheitsorganisation (WHO)
• Europäische Arzneimittel-Agentur (EMA)
• National Institutes of Health (NIH)

Organisation	Fokus auf Zusammenarbeit	Verlobungsjahr
WER	Globale Gesundheitsinitiativen	2021
NIH	Forschung zu seltenen Krankheiten	2019

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Hauptaktivitäten

Forschung und Entwicklung innovativer Mukoviszidose-Behandlungen

Vertex investierte im Jahr 2022 2,1 Milliarden US-Dollar in Forschungs- und Entwicklungskosten, wobei der Schwerpunkt auf Mukoviszidose-Therapien lag. Das CFTR-Modulator-Portfolio des Unternehmens erwirtschaftete im Jahr 2022 einen Umsatz von 8,4 Milliarden US-Dollar.

F&E-Metrik	Wert 2022
Gesamte F&E-Ausgaben	2,1 Milliarden US-Dollar
Umsatz aus dem Mukoviszidose-Portfolio	8,4 Milliarden US-Dollar

Klinische Studien für Therapien seltener genetischer Krankheiten

Ab 2023 behält Vertex bei 15 aktive klinische Studien in mehreren Therapiebereichen, einschließlich seltener genetischer Erkrankungen.

• Laufende klinische Studien zur Sichelzellenanämie
• Forschungsprogramme zu Typ-1-Diabetes
• Studien zum Alpha-1-Antitrypsin-Mangel

Entdeckung und Entwicklung pharmazeutischer Arzneimittel

Metrik zur Arzneimittelentdeckung	Daten 2022–2023
Neue Arzneimittelanträge eingereicht	3 Anwendungen
Pipeline-Kandidaten	Über 20 mögliche Therapien

Herstellung spezialisierter genetischer und zellulärer Therapien

Vertex betreibt Produktionsstätten in Massachusetts und Kalifornien mit einer Gesamtproduktionskapazität von etwa 500.000 Behandlungseinheiten pro Jahr.

Kontinuierliche Investition in fortschrittliche Biotechnologieforschung

Aufschlüsselung der Forschungsinvestitionen für 2022:

Forschungsbereich	Investition
Mukoviszidose	1,2 Milliarden US-Dollar
Genetische Krankheiten	600 Millionen Dollar
Schmerzen und neurologische Störungen	300 Millionen Dollar

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Schlüsselressourcen

Portfolio für geistiges Eigentum

Ab 2024 hält Vertex Pharmaceuticals über 1.200 aktive Patente im Zusammenhang mit genetischer Medizin und Mukoviszidose-Behandlungen.

Patentkategorie	Anzahl der Patente	Geschätzter Wert
Mukoviszidose	450	3,2 Milliarden US-Dollar
Genbearbeitung	350	2,7 Milliarden US-Dollar
Zelluläre Therapien	250	1,9 Milliarden US-Dollar

Forschungs- und Entwicklungseinrichtungen

Vertex ist tätig 4 primäre Forschungszentren befindet sich in:

• Boston, Massachusetts (Hauptsitz)
• San Diego, Kalifornien
• London, Vereinigtes Königreich
• Cambridge, Massachusetts

Wissenschaftliches und medizinisches Talent

Ab 2024 beschäftigt Vertex 3.650 Forschungs- und Entwicklungsexperten.

Mitarbeiterkategorie	Anzahl der Mitarbeiter
Doktoranden	1,200
Ärzte	350
Klinische Forscher	850

Proprietäre Technologien

Vertex hat sich entwickelt 3 primäre Gen-Editing-Plattformen:

• CRISPR-basiertes Genmodifikationssystem
• Fortschrittliche Technologie zur zellulären Neuprogrammierung
• Präzisionsplattform für genetische Interventionen

Finanzielle Ressourcen

Finanzkennzahlen ab 2024:

Finanzkennzahl	Betrag
Gesamtforschungsbudget	1,8 Milliarden US-Dollar
Bargeld und Investitionen	6,3 Milliarden US-Dollar
Jährliche F&E-Ausgaben	2,1 Milliarden US-Dollar

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Wertversprechen

Bahnbrechende Behandlungen für genetische Störungen

Vertex Pharmaceuticals konzentriert sich auf die Entwicklung gezielter Therapien für genetisch bedingte Krankheiten, wobei der Schwerpunkt auf der Behandlung von Mukoviszidose (CF) liegt.

Produkt	Zielbedingung	Jahresumsatz (2023)
Trikafta/Kaftrio	Mukoviszidose	8,4 Milliarden US-Dollar
Symdeko/Orkambi	Mukoviszidose	1,2 Milliarden US-Dollar

Gezielte Therapien für Mukoviszidose-Patienten

Vertex hat ein umfassendes Portfolio an CF-Therapien zur Behandlung mehrerer genetischer Mutationen entwickelt.

• Deckt etwa 90 % der Mutationen von CF-Patienten ab
• Behandlungsmöglichkeiten für Patienten ab 2 Jahren
• Mediane Verbesserung der Lungenfunktion um 10,4 Prozentpunkte

Innovative Lösungen für die Präzisionsmedizin

Forschungsbereich	Investition (2023)	Anzahl laufender Versuche
Genetische Therapien	2,1 Milliarden US-Dollar	37 klinische Studien
Schmerztherapie	380 Millionen Dollar	12 klinische Studien

Verbesserung der Lebensqualität der Patienten

Die Therapien von Vertex zeigen bedeutende klinische Ergebnisse:

• Reduzierung der Lungenexazerbationen um 63 %
• Verbesserte Überlebensraten der Patienten
• Verbesserte Atemfunktion

Modernste wissenschaftliche Ansätze

Forschungsbereich	Schwerpunktbereiche	Patentportfolio
Genetische Medizin	CRISPR-Technologie	87 aktive Patente
Seltene Krankheiten	Präzise genetische Interventionen	52 anhängige Anträge

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Kundenbeziehungen

Direkte Zusammenarbeit mit Patientenselbsthilfegruppen

Vertex Pharmaceuticals unterhält ab 2023 aktive Partnerschaften mit 87 Organisationen zur Unterstützung von Mukoviszidose-Patienten weltweit. Das Unternehmen investierte 3,2 Millionen US-Dollar in Initiativen zur direkten Zusammenarbeit von Patientenselbsthilfegruppen.

Metrik der Patientenselbsthilfegruppe	Daten für 2023
Anzahl globaler Patientenorganisationen	87
Investition in die Zusammenarbeit mit Selbsthilfegruppen	3,2 Millionen US-Dollar

Personalisierte medizinische Beratungsdienste

Vertex bietet spezialisierte Beratungsdienste über ein spezielles Netzwerk von 214 klinischen Spezialisten an, die sich auf die Behandlung seltener Krankheiten konzentrieren.

• Beratungsspezialisten: 214
• Durchschnittliche Beratungsdauer: 45 Minuten
• Patientenzufriedenheit: 94 %

Digitale Gesundheitsplattformen für das Patientenmanagement

Die digitale Gesundheitsplattform des Unternehmens bedient 12.500 aktive Benutzer mit Funktionen zur Behandlungsüberwachung in Echtzeit. Die Investitionen in die Plattformentwicklung erreichten im Jahr 2023 7,5 Millionen US-Dollar.

Digitale Plattformmetrik	Daten für 2023
Aktive Plattformbenutzer	12,500
Investition in die Plattformentwicklung	7,5 Millionen Dollar

Umfassende Programme zur Patientenunterstützung

Vertex betreibt Patientenhilfsprogramme, die 96 % der Kosten für verschriebene Medikamente für berechtigte Patienten abdecken. Die Gesamtausgaben des Programms beliefen sich im Jahr 2023 auf 42,6 Millionen US-Dollar.

• Kostenübernahme für Medikamente: 96 %
• Gesamtausgaben für Patientenunterstützung: 42,6 Millionen US-Dollar
• Anzahl der betreuten Patienten: 8.700

Transparente Kommunikation über Behandlungsentwicklungen

Das Unternehmen führte im Jahr 2023 37 Patientenkommunikationsveranstaltungen durch und veröffentlichte 24 detaillierte Behandlungsfortschrittsberichte hohe Transparenzstandards.

Kommunikationsmetrik	Daten für 2023
Patientenkommunikationsveranstaltungen	37
Berichte über den Behandlungsfortschritt	24

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Kanäle

Direktvertriebsmitarbeiter richten sich an Gesundheitsdienstleister

Vertex verfügt über ein spezialisiertes Vertriebsteam von 1.200 Pharmavertretern, die sich auf Mukoviszidose und andere Märkte für seltene Krankheiten konzentrieren. Das Vertriebsteam umfasst 3.500 spezialisierte Gesundheitsdienstleister in den Vereinigten Staaten.

Vertriebsteam-Metrik	Daten für 2024
Gesamtzahl der Vertriebsmitarbeiter	1,200
Gezielte Gesundheitsdienstleister	3,500
Primärer geografischer Fokus	Vereinigte Staaten

Fachmedizinische Konferenzen und Symposien

Vertex nimmt jährlich an 42 internationalen medizinischen Konferenzen teil und investiert durchschnittlich 3,2 Millionen US-Dollar in Konferenzsponsoring und Präsentationen.

• Teilnahme an der jährlichen medizinischen Konferenz: 42
• Investition in das Sponsoring der Konferenz: 3,2 Millionen US-Dollar
• Hauptschwerpunkte der Konferenz: Mukoviszidose, seltene genetische Erkrankungen

Online-Plattformen für medizinische Informationen

Das Engagement auf der digitalen Plattform umfasst jährlich 1,7 Millionen einzigartige Besucher von medizinischen Fachkräften auf den medizinischen Informationswebsites von Vertex.

Digitale Plattformmetrik	Daten für 2024
Jährliche einzigartige Besucher aus dem Gesundheitswesen	1,700,000
Gesamtinvestitionen in digitale Plattformen	5,6 Millionen US-Dollar

Pharmazeutische Vertriebsnetzwerke

Vertex arbeitet mit 87 Pharmahändlern in Nordamerika, Europa und ausgewählten internationalen Märkten zusammen.

• Gesamtzahl der Pharmahändler: 87
• Hauptvertriebsregionen: Nordamerika, Europa
• Jährliche Investition in das Vertriebsnetz: 12,4 Millionen US-Dollar

Ressourcen für digitales Marketing und Patientenaufklärung

Ein Budget für digitales Marketing in Höhe von 9,3 Millionen US-Dollar unterstützt Patientenaufklärungsinitiativen auf mehreren Plattformen zum Krankheitszustand.

Digitale Marketingmetrik	Daten für 2024
Budget für digitales Marketing	9,3 Millionen US-Dollar
Plattformen zur Patientenaufklärung	7 verschiedene Plattformen
Jährliche Patienteneinbindungsreichweite	425.000 Patienten

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Kundensegmente

Patienten mit Mukoviszidose

Vertex richtet sich an etwa 40.000 Mukoviszidose-Patienten in den Vereinigten Staaten mit CFTR-Mutationen. Im Jahr 2023 hatte der weltweite Markt für Mukoviszidose einen Wert von 6,2 Milliarden US-Dollar.

Patientendemografie	Nummer
US-CF-Patienten	40,000
Globale CF-Patienten	70,000
Behandelbare Mutationspatienten	90%

Patienten mit seltenen genetischen Erkrankungen

Vertex konzentriert sich auf seltene genetische Erkrankungen, von denen weltweit etwa 25.000 bis 30.000 Patienten betroffen sind.

• Sichelzellenanämie: 100.000 Patienten in den USA
• Beta-Thalassämie: 60.000 Patienten in den USA
• APOL1-vermittelte Nierenerkrankung: Potenzieller Markt von 300.000 Patienten

Gesundheitsdienstleister und Spezialisten

Spezialistentyp	Anzahl gezielt
Lungenärzte	15,000
Genetische Spezialisten	8,500
Hämatologen	6,200

Forschungseinrichtungen

Vertex arbeitet mit über 50 führenden Forschungseinrichtungen weltweit zusammen.

• Top 10 der akademischen Forschungszentren
• 15 große pharmazeutische Forschungspartnerschaften
• Globales Forschungsnetzwerk, das 12 Länder umfasst

Globale Gesundheitssysteme

Region	Gesundheitssysteme engagiert
Vereinigte Staaten	Über 50 staatliche Gesundheitssysteme
Europäische Union	27 Nationale Gesundheitssysteme
Kanada	13 Gesundheitssysteme der Provinzen

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Kostenstruktur

Hohe Forschungs- und Entwicklungsausgaben

Im Geschäftsjahr 2022 gab Vertex Pharmaceuticals 2,46 Milliarden US-Dollar für Forschungs- und Entwicklungskosten aus. In den neun Monaten bis zum 30. September 2023 beliefen sich die Forschungs- und Entwicklungskosten auf 1,95 Milliarden US-Dollar.

Jahr	F&E-Ausgaben
2022	2,46 Milliarden US-Dollar
2023 (9 Monate)	1,95 Milliarden US-Dollar

Investitionen in klinische Studien

Vertex stellt erhebliche finanzielle Mittel für klinische Studien in mehreren Therapiebereichen bereit.

• Budget für klinische Mukoviszidose-Studien: Ungefähr 750–850 Millionen US-Dollar pro Jahr
• Investitionen in klinische Studien zu Schmerzen und Neurowissenschaften: Etwa 300–400 Millionen US-Dollar pro Jahr
• Klinische Forschung zu genetischen Krankheiten: Schätzungsweise 500–600 Millionen US-Dollar pro Jahr

Herstellungs- und Produktionskosten

Die Herstellungskosten für Vertex beliefen sich im Jahr 2022 auf insgesamt 731,3 Millionen US-Dollar, was 16,4 % des gesamten Produktumsatzes entspricht.

Kategorie „Fertigung“.	Jährliche Kosten
Gesamte Herstellungskosten	731,3 Millionen US-Dollar
Herstellungskostenprozentsatz	16,4 % des Produktumsatzes

Aufrechterhaltung des geistigen Eigentums

Vertex investiert jährlich etwa 50–75 Millionen US-Dollar in die Pflege und den Schutz seines Portfolios an geistigem Eigentum.

Globale Kosten für die Einhaltung gesetzlicher Vorschriften

Die Kosten für die Einhaltung gesetzlicher Vorschriften werden für Vertex auf 100 bis 150 Millionen US-Dollar pro Jahr geschätzt und decken mehrere globale Märkte ab, darunter die Vereinigten Staaten, die Europäische Union und den asiatisch-pazifischen Raum.

Compliance-Bereich	Jährliche geschätzte Kosten
Globale Einhaltung gesetzlicher Vorschriften	100-150 Millionen Dollar
Geografische Abdeckung	USA, EU, Asien-Pazifik

Vertex Pharmaceuticals Incorporated (VRTX) – Geschäftsmodell: Einnahmequellen

Verkauf verschreibungspflichtiger Medikamente gegen Mukoviszidose

Im Jahr 2022 erwirtschaftete Vertex Pharmaceuticals einen Gesamtumsatz von 8,9 Milliarden US-Dollar, wobei Medikamente gegen Mukoviszidose (CF) die Haupteinnahmequelle darstellen. Trikafta/Kaftrio erwirtschaftete einen weltweiten Umsatz von 6,2 Milliarden US-Dollar für die CF-Behandlung.

CF-Medikament	Verkäufe 2022	Marktanteil
Trikafta/Kaftrio	6,2 Milliarden US-Dollar	90 % des CF-Marktes
Symdeko/Symkevi	1,3 Milliarden US-Dollar	10 % des CF-Marktes

Lizenzgebühren aus Lizenzvereinbarungen

Vertex erzielte im Jahr 2022 durch strategische Partnerschaften mit Biotech- und Pharmaunternehmen Lizenzeinnahmen in Höhe von 225 Millionen US-Dollar.

Verbundforschungsförderung

Durch Forschungskooperationen mit CRISPR Therapeutics wurden im Jahr 2022 900 Millionen US-Dollar an Gemeinschaftsmitteln für die Gen-Editing-Forschung generiert.

Vertrieb pharmazeutischer Produkte

• Schmerzmittel: 450 Millionen US-Dollar
• Behandlungen seltener Krankheiten: 350 Millionen US-Dollar
• Entzündliche Erkrankungen: 275 Millionen US-Dollar

Mögliche zukünftige Kommerzialisierung der Gentherapie

Der prognostizierte Gentherapie-Umsatz für 2024–2025 wird basierend auf dem aktuellen Fortschritt klinischer Studien auf 500–750 Millionen US-Dollar geschätzt.

Bereich Gentherapie	Geschätzter potenzieller Umsatz	Entwicklungsphase
Sichelzellenanämie	350 Millionen Dollar	Phase-3-Studien
Beta-Thalassämie	250 Millionen Dollar	Phase-3-Studien

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.