Vertex Pharmaceuticals

NASDAQ:VRTXBiotech

Boston, 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.

Pipeline Programs↗

Distinct drugs in development (Preclinical–Phase 3)

Indications↗

Distinct diseases across all programs

248

Clinical Trials↗

All sponsored trials (all statuses)

Orange Book Patents

Across 3 drugs with upcoming expirations

Pipeline

Pipeline Snapshot

By Phase

18 drugs in development

Phase 36

Phase 27

Phase 17

Preclinical2

By Therapeutic Area

top 6

Rare Disease

836%

Nephrology

314%

Metabolic & Endocrine

314%

Neurology

29%

Hematology

15%

Infectious Disease

15%

R&D Momentum

65/100

Pipeline Velocity

34/100

Trial Completion Rate

90/100

Pipeline Concentration

20/100

Pipeline Freshness

78/100

Strategic Signals

↗Active in Hematology but thin pipeline — potential licensing opportunity

Activity

Recent Activity

Last 14 days

Jul 2, 2026

Active, not recruiting

A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia

Jul 2, 2026

Active, not recruiting

A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease

Jul 1, 2026

Enrolling by invitation

An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type I

Jul 1, 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

Jul 1, 2026

Recruiting

A Phase 1/2/3 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-880 in Subjects Who Have Type 1 Diabetes Mellitus With Impaired Hypoglycemic Awareness and Severe Hypoglycemia

Jul 1, 2026

Active, not recruiting

A Phase 1, Open-label Drug-drug Interaction Study to Evaluate the Effects of Efavirenz on the Pharmacokinetics of Suzetrigine in Healthy Subjects

Earlier

Jun 30, 2026TrialA 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

Jun 30, 2026TrialA Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study With a Long-term Extension of Povetacicept in Adults With Generalized Myasthenia Gravis

4 more events

Jun 26, 2026TrialA 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

Feb 23, 2026TrialDose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants

Feb 13, 2026TrialEvaluation of the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female Participants

Jan 12, 2026TrialEffect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants

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

Rare DiseaseHematologyOtherInfectious DiseaseOncologyRespiratoryMetabolic & EndocrineGastroenterologyMusculoskeletalNeurologyCardiovascularImmunologyNephrology

Patent Expirations

8 patent groups across drugs · Source: FDA Orange Book

DrugExpires

Kalydeco3 patentsJul 6, 2026 Kalydeco6 patentsJan 6, 2027 KalydecoFeb 5, 2028 IncivekMay 30, 2028 KalydecoAug 13, 2029 Kalydeco3 patentsFeb 13, 2030 Kalydeco7 patentsFeb 27, 2033 JournavxDec 4, 2040

Pipeline

Drug Pipeline

Therapeutic Area

Preclinical

Phase 1

Phase 2

Phase 3

Approved

Hematology(1 drug)

—

Hematology(1 drug)

Infectious Disease(1 drug)

—

Infectious Disease(1 drug)

Metabolic & Endocrine(3 drugs)

—

Metabolic & Endocrine(3 drugs)

Nephrology(3 drugs)

—

Nephrology(3 drugs)

Neurology(2 drugs)

—

Neurology(2 drugs)

Rare Disease(8 drugs)

—

Rare Disease(8 drugs)

Respiratory(1 drug)

—

Respiratory(1 drug)

Unknown / Other(3 drugs)

—

Unknown / Other(3 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)