Vertex Pharmaceuticals

Overview

Vertex Pharmaceuticals is a global biotechnology company focused on developing and commercializing transformative medicines for people with serious diseases, with an emphasis on specialty markets. The company combines marketed products with ongoing pipeline programs and strategic collaborations to drive serial innovation.

Marketed products and lead therapeutic areas

Cystic Fibrosis (CF)

• Approved CFTR modulators:
  • KALYDECO (ivacaftor)
  • ORKAMBI (lumacaftor/ivacaftor)
  • SYMDEKO / SYMKEVI (tezacaftor/ivacaftor and ivacaftor)
  • TRIKAFTA / KAFTRIO (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
  • ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor)
• Market presence:
  • Nearly three quarters of the ~97,000 people with CF in the U.S., Europe, Australia, and Canada are on Vertex CF medicines.
  • Estimated CF population across target markets: ~112,000.
  • CF medicines are reimbursed or accessible in more than 60 countries across six continents.
  • ALYFTREK is a once-daily triple therapy and has demonstrated non-inferiority to TRIKAFTA on certain endpoints.

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

• CASGEVY (exagamglogene autotemcel): a CRISPR/Cas9-based, ex vivo gene-edited HSPC therapy for severe SCD and TDT.
  • Approved in the U.S. and multiple other geographies (Europe, Canada, Middle East).
  • Agreements with national and regional payors covering more than 275 million lives.
  • 2025 activity: ~300 patients treated for SCD/TDT; ~147 cell collections; ~64 infusions.
  • Global access via authorized treatment centers (ATCs) and an expanding payer network.

Acute Pain

• JOURNAVX (suzetrigine): a selective NaV1.8 pain signal inhibitor.
  • FDA approved in January 2025 for moderate-to-severe acute pain in adults.
  • 2025 performance: over 550,000 prescriptions written/filled across hospital and retail channels.
  • Commercial access includes all three U.S. national pharmacy benefit managers; as of January 2026, coverage for JOURNAVX is available to more than 200 million individuals across commercial and government payors, and 21 states include coverage via Medicaid.

Pipeline and research-stage programs (highlights)

• IgA Nephropathy (IgAN) and other B cell–mediated diseases
  • Povetacicept (dual BAFF/APRIL inhibitor): Phase 3 (RAINIER) in IgAN; rolling BLA submitted to FDA in late 2025; interim data expected in H1 2026 with potential for accelerated approval if interim results are positive.
  • PRIME designation in the EU and Breakthrough Therapy designation in the U.S. for povetacicept in IgAN and pMN.
  • Adaptive Phase 2/3 program in primary membranous nephropathy (pMN); Phase 2 portion expected to complete and Phase 3 to begin mid-2026.
• APOL1-mediated kidney disease (AMKD)
  • Inaxaplin (APOL1 inhibitor): Phase 2/3 (AMPLITUDE) ongoing; interim analysis targeting 48 weeks of treatment, with data anticipated late 2026 or early 2027. Expanded development (AMPLIFIED) planned for additional AMKD populations.
• Pain programs
  • Suzetrigine: two Phase 3 trials for diabetic peripheral neuropathic pain (DPN); FDA Breakthrough Therapy designation for DPN.
  • VX-993: evaluated for acute pain and under evaluation in Phase 2; not advanced as monotherapy following Phase 2 results.
  • Continued exploration of additional NaV1.8 and NaV1.7 inhibitors.
• Type 1 Diabetes (T1D)
  • Zimislecel: allogeneic, stem-cell derived islet cell replacement therapy.
    • Completed enrollment in a Phase 1/2/3 trial; dosing temporarily paused for an internal manufacturing analysis.
    • Published clinical data (NEJM 2025) support potential transformative benefit.
    • Designations include FDA RMAT and Fast Track, EMA PRIME, Saudi Arabia Breakthrough, and UK Innovative Licensing and Access Pathway.
  • VX-264 (encapsulated zimislecel) program discontinued after phase results.
  • VX-522 (CFTR mRNA, collaboration with Moderna): nebulized therapy to restore CFTR in patients lacking full-length CFTR; dosing in Phase 1/2 with data expected in 2H 2026.
  • VX-670 (DM1 program, collaboration with Entrada Therapeutics): GALILEO program ongoing (Phase 1/2) for intracellular antisense oligonucleotide delivery in DM1.
• Autosomal Dominant Polycystic Kidney Disease (ADPKD)
  • VX-407: first-in-class small molecule corrector targeting PKD1 variants (up to ~10% of ADPKD patients).
  • AGLOW: Phase 2 proof-of-concept trial ongoing; enrollment expected to complete by end of 2026.
• Myotonic Dystrophy Type 1 (DM1)
  • VX-670 in the GALILEO program; additional preclinical approaches in development.
• Generalized Myasthenia Gravis (gMG)
  • Povetacicept being prepared for a Phase 2 dose-ranging proof-of-concept study in 1H 2026.

Strategic transactions and collaborations

• Acquisitions and in-licenses
  • Alpine Immune Sciences (2024): acquired for approximately $5.0 billion; povetacicept is a core asset across IgAN, pMN, and gMG.
  • Semma Therapeutics (2019): foundation for the T1D islet cell therapy program.
• Collaborations and licensing
  • CRISPR Therapeutics: CASGEVY co-development/co-commercialization; Vertex leads global development and commercialization; profits and losses split 60/40 (Vertex/CRISPR).
  • CRISPR licensing for CF targets and other programs; ongoing patent matters involving Broad/Charpentier.
  • Moderna: collaboration on VX-522 (CFTR mRNA).
  • Entrada Therapeutics: collaboration on VX-670 (DM1).
• Regional licensing for povetacicept (2025)
  • Zai Lab: mainland China, Hong Kong, Macau, Taiwan, and Singapore.
  • Ono Pharmaceutical: Japan and South Korea.

Manufacturing and operations

• In-house small-molecule manufacturing capabilities in Boston and certain production stages for CF therapies.
• Reliance on third-party contract manufacturers (CMOs) for many components, including CFTR modulators, povetacicept supply, and CASGEVY manufacturing.
• Expanded cell therapy manufacturing capabilities in the Boston area; strategic agreement with Lonza to support T1D cell therapy manufacturing.
• Complex global supply chains for biologics, cell therapies, and gene-editing products, with emphasis on cGMP compliance and supplier qualification.
• Ongoing efforts in pipeline-to-production scaling, capacity planning, and multi-facility coordination for CASGEVY and other products.

Intellectual property

• Large portfolio of U.S. and international patents and applications covering CFTR modulators, povetacicept, zimislecel, VX-522, VX-993, VX-407, VX-670, and other assets.
• U.S. Orange Book listings include multiple patents for KALYDECO, ORKAMBI, SYMDEKO/SYMKEVI, TRIKAFTA/KAFTRIO, and ALYFTREK.
• CASGEVY IP includes Vertex-owned patents and CRISPR-related licenses; litigation involving ToolGen is ongoing.
• Regulatory exclusivities (for example, 12-year U.S. BLA exclusivity) supplement patent protection and support market position.

Customers, patients, and access

• Payor and patient access programs are central to commercialization strategy.
• CASGEVY payor reach covers more than 275 million lives globally.
• JOURNAVX coverage is available to over 200 million covered lives in the U.S., with 21 states covering via Medicaid.
• Public and private payer engagement, formulary placement, and reimbursement discussions are core elements of pricing and market access.

People and culture

• Approximately 6,400 employees as of December 31, 2025.
  • U.S.-based employees: ~5,200
  • International employees: ~1,200
• No U.S. employees are covered by a collective bargaining agreement; a small number of international employees are covered.

Geographic and regulatory context

• Operates in a global regulatory environment (FDA, EMA, and other international regulators).
• Pricing and reimbursement are primary drivers of revenue performance.
• Intellectual property and regulatory exclusivities are central to sustaining product differentiation and market position.

Business model summary

Vertex develops and commercializes medicines across cystic fibrosis, SCD/TDT, and acute pain, while advancing a pipeline across additional serious diseases. The company combines internal discovery with external collaborations, acquisitions, and licensing to access new technologies and accelerate development. Investments in manufacturing and supply chain capabilities support multiple modalities, including small molecules, biologics, and cell/gene therapies. Patient access and payer arrangements are integral to commercialization and revenue strategy.