Vertex Pharmaceuticals
NASDAQ:VRTXBiotechBoston, Massachusetts, United States · Est. 1989 · 2,400 employees
Vertex Pharmaceuticals is a global biotechnology company that discovers, develops, and commercializes innovative therapies for serious diseases, with a primary focus on cystic fibrosis and other rare genetic conditions.
17
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
13
Indications↗
Distinct diseases across all programs
267
Clinical Trials↗
All sponsored trials (all statuses)
23
Orange Book Patents
Across 3 drugs with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
17 drugs in development
Phase 36
Phase 24
Phase 18
Preclinical1
By Therapeutic Area
top 5
Rare Disease
523%
Nephrology
523%
Metabolic & Endocrine
314%
Neurology
29%
Hematology
15%
R&D Momentum
67/100
Pipeline Velocity
41/100
Trial Completion Rate
90/100
Pipeline Concentration
21/100
Pipeline Freshness
78/100
Strategic Signals
- ↗Active in Hematology but thin pipeline — potential licensing opportunity
Activity
Recent Activity
Last 14 days
Sep 19, 2026
Recruiting
A Phase 2b/3 Adaptive, Randomized, Active-controlled Study Evaluating the Efficacy, Safety, and Tolerability of Povetacicept Versus Calcineurin Inhibitor in the Treatment of Primary Membranous Nephropathy
Sep 17, 2026
Completed
A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
Sep 17, 2026
Active, not recruiting
A Phase 2a, Open-label, Single-arm Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-407 in Subjects With Autosomal Dominant Polycystic Kidney Disease Who Have a Subset of PKD1 Gene Variants
Sep 15, 2026
Completed
Open-Label Study to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Povetacicept in Subjects With Autoimmune Cytopenias (RUBY-4)
Sep 12, 2026
Recruiting
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study With a Long-term Extension of Povetacicept in Adults With Generalized Myasthenia Gravis
Sep 11, 2026
Active, not recruiting
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus
Sep 11, 2026
Active, not recruiting
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-121/TEZ/D-IVA Combination Therapy in Subjects With Cystic Fibrosis
Sep 11, 2026
Active, not recruiting
A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes
Sep 10, 2026
Active, not recruiting
A Study to Determine the Prevalence and Clinical Characteristics Associated With PKD1 Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Sep 10, 2026
Recruiting
A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease
Sep 10, 2026
Completed
A Phase 1, Open-label, Single-dose Study Evaluating the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female Subjects
Details
Company Info
Company Overview
$67.0B
Mkt Cap
$7.7B
Revenue
—
Cash
CEOReshma Kewalramani
Founded1989
Employees2,400
HQBoston, Massachusetts, United States
ExchangeNASDAQ:VRTX
Websitevrtx.com
LinkedInCompany Page
Therapeutic Focus
Infectious DiseaseOtherHematologyGastroenterologyNeurologyMusculoskeletalCardiovascularRare DiseaseRespiratoryImmunologyMetabolic & EndocrineNephrologyOncology
Pipeline
Drug Pipeline
Therapeutic Area
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Hematology(1 drug)
—
—
—
1
—
Hematology(1 drug)
Metabolic & Endocrine(3 drugs)
—
—
1
2
—
Metabolic & Endocrine(3 drugs)
Nephrology(5 drugs)
1
3
1
—
—
Nephrology(5 drugs)
Neurology(2 drugs)
—
—
2
—
—
Neurology(2 drugs)
Rare Disease(5 drugs)
—
2
—
3
—
Rare Disease(5 drugs)
Unknown / Other(6 drugs)
—
6
—
—
—
Unknown / Other(6 drugs)
Regulatory
Regulatory Timeline
20111 event
FDAMay 23
Incivek — INCIVEK
Application NDA201917 (NDA)
20121 event
FDAJan 31
Kalydeco — KALYDECO
Application NDA203188 (NDA)
20231 event
FDAMay 3
Kalydeco — KALYDECO
Application NDA207925 (NDA)
20251 event
FDAJan 30
Journavx — JOURNAVX
Application NDA219209 (NDA)
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)