VP

Vertex Pharmaceuticals(VRTX)

BOSTON, MA
Biotechnology1 H-1B visas (FY2023)
Cystic FibrosisPainSickle Cell DiseaseRare Diseases
Funding Stage
PUBLIC
Employees
10,400
Open Jobs
295

Products & Portfolio (9)

1 discontinued product not shown

ALYFTREK
vanzacaftor, tezacaftor, and deutivacaftor
Growth
ORAL · TABLET
Chloride Channel Activation Potentiators
cystic fibrosis (CF) in patients aged 6 yearsolderanother responsive mutation in the CFTR gene
2024
0
JOURNAVX
suzetrigine
Growth
ORAL · TABLET
Sodium Channel Antagonists
2025
0
KALYDECO
ivacaftor
Peak
ORAL · GRANULE
Chloride Channel Activation Potentiators
2015
0
KALYDECO
ivacaftor
Peak
ORAL · TABLET
Chloride Channel Activation Potentiators
2012
8
ORKAMBI
lumacaftor and ivacaftor
Peak
ORAL · TABLET
Chloride Channel Activation Potentiators
2015
0
ORKAMBI
lumacaftor and ivacaftor
Peak
ORAL · GRANULE
Chloride Channel Activation Potentiators
2018
0
SYMDEKO (COPACKAGED)
tezacaftor and ivacaftor
Peak
ORAL · TABLET
(including F508del-CFTR) to increase the amount of mature CFTR protein delivered to the cell surface. Ivacaftor is a CFTR potentiator that facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein at the cell surface. For ivacaftor to function CFTR protein must be present at the cell surface. Ivacaftor can potentiate the CFTR protein delivered to the cell surface by tezacaftor, leading to a further enhancement of chloride transport than either agent alone. The combined effect of tezacaftor and ivacaftor is increased quantity and function of CFTR at the cell surface, resulting in increases in chloride transport. CFTR Chloride Transport Assay in Fischer Rat Thyroid (FRT) cells expressing mutant CFTR The chloride transport response of mutant CFTR protein to tezacaftor/ivacaftor was determined in Ussing chamber electrophysiology studies using a panel of FRT cell lines transfected with individual CFTR mutations. Tezacaftor/ivacaftor increased chloride transport in FRT cells expressing CFTR mutations that result in CFTR protein being delivered to the cell surface. The in vitro chloride transport response threshold was designated as a net increase of at least 10% of normal over baseline because it is predictive or reasonably expected to predict clinical benefit. For individual mutations, the magnitude of the net change over baseline in CFTR-mediated chloride transport in vitro is not correlated with the magnitude of clinical response. Note that splice site mutations cannot be studied in the FRT assay. Table 6 lists responsive CFTR mutations based on (1) a clinical FEV 1 response and/or (2) in vitro data in FRT cells, indicating that tezacaftor/ivacaftor increases chloride transport to at least 10% of normal over baseline. CFTR gene mutations that are not responsive to ivacaftor alone are not expected to respond to SYMDEKO except for F508del homozygotes. Table 6: List of CFTR Gene Mutations that Produce CFTR Protein and are Responsive to SYMDEKO 546insCTA E92K G576A L346P R117G S589N 711+3A→G Clinical data for these mutations in Clinical Studies [see and ] . E116K G576A;R668C Complex/compound mutations where a single allele of the CFTR gene has multiple mutations; these exist independent of the presence of mutations on the other allele. L967S R117H S737F 2789+5G→A E193K G622D L997F R117L S912L 3272-26A→G E403D G970D L1324P R117P S945L 3849+10kbC→T E588V G1069R L1335P R170H S977F A120T E822K G1244E L1480P R258G S1159F A234D E831X G1249R M152V R334L S1159P A349V F191V G1349D M265R R334Q S1251N A455E F311del H939R M952I R347H S1255P A554E F311L H1054D M952T R347L T338I A1006E F508C H1375P P5L R347P T1036N A1067T F508C;S1251N I148T P67L R352Q T1053I D110E F508del A patient must have two copies of the F508del mutation or at least one copy of a responsive mutation presented in Table 6 to be indicated. I175V P205S R352W V201M D110H F575Y I336K Q98R R553Q V232D D192G F1016S I601F Q237E R668C V562I D443Y
cystic fibrosis (CF) in patients aged 6 yearsolder
2018
0
TRIKAFTA (COPACKAGED)
elexacaftor, tezacaftor, and ivacaftor
Peak
ORAL · TABLET
(including F508del-CFTR) to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Ivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface. The combined effect of elexacaftor, tezacaftor and ivacaftor is increased quantity and function of CFTR at the cell surface, resulting in increased CFTR activity as measured both by CFTR mediated chloride transport in vitro and by sweat chloride in patients with CF. CFTR Chloride Transport Assay in Fischer Rat Thyroid Cells Expressing Mutant CFTR Protein Effects of elexacaftor/tezacaftor/ivacaftor on chloride transport for mutant CFTR proteins was determined in Ussing chamber electrophysiology studies using a panel of Fischer Rat Thyroid (FRT) cell lines stably expressing individual mutant CFTR protein. Elexacaftor/tezacaftor/ivacaftor increased chloride transport in FRT cells expressing CFTR mutations, as identified in Table 6. The threshold that the treatment-induced increase in chloride transport must exceed for the mutant CFTR protein to be considered responsive is ≥10% of normal over baseline. This threshold was used because it is expected to predict clinical benefit. For individual mutations, the magnitude of the net change over baseline in CFTR-mediated chloride transport in vitro is not correlated with the magnitude of clinical response. CFTR Chloride Transport Assay in Human Bronchial Epithelial Cells Expressing Mutant CFTR Protein Homozygous and heterozygous N1303K -Human Bronchial Epithelial (HBE) cells showed greater chloride transport in the presence of elexacaftor/tezacaftor/ivacaftor than F508del/F508del -HBE cells treated with tezacaftor/ivacaftor (which has shown clinical benefit in people homozygous for F508del) . Patient Selection Select patients 2 years of age and older for treatment of CF with TRIKAFTA based on the presence of at least one F508del mutation or another responsive mutation in the CFTR gene (see ) [see ] . Table 6 lists CFTR mutations responsive to TRIKAFTA based on clinical response and/or in vitro data in FRT or HBE cells or based on extrapolation of efficacy. Table 6: List of CFTR Gene Mutations Responsive to TRIKAFTA Mutations responsive to TRIKAFTA based on clinical data Clinical data obtained from Trials 1, 2, and 5. 2789+5G→A D1152H This mutation is also predicted to be responsive by FRT assay. L206W R1066H S945L 3272-26A→G F508del L997F R117C T338I 3849+10kbC→T G85E M1101K R347H V232D A455E L1077P P5L R347P Mutations responsive to TRIKAFTA based on in vitro data The N1303K mutation is predicted to be responsive by HBE assay. All other mutations predicted to be responsive with in vitro data are supported by FRT assay. N1303K F200I I1139V P574H S1045Y 1507_1515del9 F311del I125T P67L S108F 2183A→G F311L I1269N P750L S1118F 3141del9 F508C I1366N Q1291R S1159F 546insCTA F508C;S1251N I148N Q1313K S1159P A1006E F575Y I148T Q237E S1235R A1067P F587I I175V Q237H S1251N A1067T G1047R I
cystic fibrosis (CF) in patients aged 2 yearsoldera mutation in the CFTR gene
2019
0
TRIKAFTA (COPACKAGED)
elexacaftor, tezacaftor, and ivacaftor
Growth
ORAL · GRANULES
(including F508del-CFTR) to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Ivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface. The combined effect of elexacaftor, tezacaftor and ivacaftor is increased quantity and function of CFTR at the cell surface, resulting in increased CFTR activity as measured both by CFTR mediated chloride transport in vitro and by sweat chloride in patients with CF. CFTR Chloride Transport Assay in Fischer Rat Thyroid Cells Expressing Mutant CFTR Protein Effects of elexacaftor/tezacaftor/ivacaftor on chloride transport for mutant CFTR proteins was determined in Ussing chamber electrophysiology studies using a panel of Fischer Rat Thyroid (FRT) cell lines stably expressing individual mutant CFTR protein. Elexacaftor/tezacaftor/ivacaftor increased chloride transport in FRT cells expressing CFTR mutations, as identified in Table 6. The threshold that the treatment-induced increase in chloride transport must exceed for the mutant CFTR protein to be considered responsive is ≥10% of normal over baseline. This threshold was used because it is expected to predict clinical benefit. For individual mutations, the magnitude of the net change over baseline in CFTR-mediated chloride transport in vitro is not correlated with the magnitude of clinical response. CFTR Chloride Transport Assay in Human Bronchial Epithelial Cells Expressing Mutant CFTR Protein Homozygous and heterozygous N1303K -Human Bronchial Epithelial (HBE) cells showed greater chloride transport in the presence of elexacaftor/tezacaftor/ivacaftor than F508del/F508del -HBE cells treated with tezacaftor/ivacaftor (which has shown clinical benefit in people homozygous for F508del) . Patient Selection Select patients 2 years of age and older for treatment of CF with TRIKAFTA based on the presence of at least one F508del mutation or another responsive mutation in the CFTR gene (see ) [see ] . Table 6 lists CFTR mutations responsive to TRIKAFTA based on clinical response and/or in vitro data in FRT or HBE cells or based on extrapolation of efficacy. Table 6: List of CFTR Gene Mutations Responsive to TRIKAFTA Mutations responsive to TRIKAFTA based on clinical data Clinical data obtained from Trials 1, 2, and 5. 2789+5G→A D1152H This mutation is also predicted to be responsive by FRT assay. L206W R1066H S945L 3272-26A→G F508del L997F R117C T338I 3849+10kbC→T G85E M1101K R347H V232D A455E L1077P P5L R347P Mutations responsive to TRIKAFTA based on in vitro data The N1303K mutation is predicted to be responsive by HBE assay. All other mutations predicted to be responsive with in vitro data are supported by FRT assay. N1303K F200I I1139V P574H S1045Y 1507_1515del9 F311del I125T P67L S108F 2183A→G F311L I1269N P750L S1118F 3141del9 F508C I1366N Q1291R S1159F 546insCTA F508C;S1251N I148N Q1313K S1159P A1006E F575Y I148T Q237E S1235R A1067P F587I I175V Q237H S1251N A1067T G1047R I
cystic fibrosis (CF) in patients aged 2 yearsoldera mutation in the CFTR gene
2023
0

Pipeline & Clinical Trials

N/A
Clinical Trials (1)
NCT03795363Orkambi Treatment in 2 to 5 Year Old Children With CF
N/A
N/A
Clinical Trials (1)
NCT03783286Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects
N/A
Standard OGTT
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT03227094Simplification of CF-related Diabetes Screening at Home
N/A
Questionnaires
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT03659214Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults
N/A
Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT06506773Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
N/A
telaprevir
Hepatitis C
N/A
Clinical Trials (1)
NCT00916474Virology Follow up Study in Subjects Previously Treated With Telaprevir
N/A
MuCopilot mobile application
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT06147778Performances and Safety of MuCopilot, a Digital Tool for the Unsupervised Objective Assessment of Cystic Fibrosis
N/A
Clinical Trials (1)
NCT01381289VX-770 Expanded Access Program
N/A
A Decentralized Study to Evaluate Physical Activity and Cough Frequency Using Wearable Technology in
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT04923464A Decentralized Study to Evaluate Physical Activity and Cough Frequency Using Wearable Technology in Cystic Fibrosis
N/A
N/A
Clinical Trials (1)
NCT02445053Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
N/A
tezacaftor/ivacaftor
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT03278314Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis
N/A
Longitudinal Assessment of Exercise Capacity and Vascular Function in Patients With CF
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT03338595Longitudinal Assessment of Exercise Capacity and Vascular Function in Patients With CF
N/A
A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibros
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT02722057A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients
N/A
Effect of Lumacaftor/Ivacaftor in Children With Cystic Fibrosis Homozygote for F508del on Small Airw
Cystic Fibrosis in Children
N/A
Clinical Trials (1)
NCT04138589Effect of Lumacaftor/Ivacaftor in Children With Cystic Fibrosis Homozygote for F508del on Small Airway Function
N/A
ELX/TEZ/IVA
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT04058210VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)
N/A
Ophthalmologic examinations
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT01863238An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic Fibrosis
N/A
Clinical Trials (1)
NCT00923728A Phase I Trial of AVN944, an IMPDH Inhibitor, in Adults With Advanced Stage Solid Tumors
N/A
Energy Balance and Weight Gain With Ivacaftor Treatment
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT02141464Energy Balance and Weight Gain With Ivacaftor Treatment
N/A
ELX/TEZ/IVA
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT04702360ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
N/A
Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disea
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
N/A
Clinical Trials (1)
NCT06747572Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease
N/A
Magnetic Resonance Imaging
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT04618185Gut Imaging for Function & Transit in CF - GIFT-CF 3
N/A
Connected Devices for 3 months
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT03304028MUCOviscidose EXacerbation Outils Connectés Education Thérapeutique
N/A
Rectal Biopsy
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT04254705Organoid Study R334W
N/A
Gastrointestinal Study at Orkambi Therapy in CF Patients
Cystic Fibrosis
N/A
Clinical Trials (1)
NCT03859531Gastrointestinal Study at Orkambi Therapy in CF Patients
N/A
Phase 1
Clinical Trials (1)
NCT00623649Safety,Tolerability and Pharmacokinetics of Multiple Ascending Doses of VCH 916 in Subjects With Chronic Hep C Infection
Phase 1
VNZ/TEZ/D-IVA
Cystic Fibrosis
Phase 1
Clinical Trials (1)
NCT06299709A Study To Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Vanzacaftor/Tezacaftor/Deutivacaftor(VNZ/TEZ/D-IVA)
Phase 1
Clinical Trials (1)
NCT07074327Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy Participants
Phase 1
Suzetrigine
Pain
Phase 1
Clinical Trials (1)
NCT06336096A Study to Evaluate the Relative Bioavailability and Food Effect of a New Tablet Formulation and Strength of Suzetrigine
Phase 1
Povetacicept
Healthy Volunteers
Phase 1
Clinical Trials (1)
NCT07010406A Study to Evaluate the Relative Bioavailability of Povetacicept Formulations and Bioequivalence of Povetacicept Presentations
Phase 1
Clinical Trials (1)
NCT05579431A Phase 1, First-in-human Study of VX-634
Phase 1
VX-993
Pain
Phase 1
Clinical Trials (1)
NCT06394167A Phase 1 Dose Escalation Study of Intravenous VX-993 in Healthy Adults
Phase 1
Clinical Trials (1)
NCT01208285Study of VX-770 in Subjects With Moderate Hepatic Impairment and in Matched Healthy Subjects
Phase 1
VX-993
Pain
Phase 1
Clinical Trials (1)
NCT06392659A Phase 1, Open-label Study Evaluating the Pharmacokinetics and Drug-drug Interaction of VX-993 in Healthy Adults
Phase 1
VX-985 or matching placebo
Chronic Hepatitis C
Phase 1
Clinical Trials (1)
NCT01144936Study of VX-985 in Subjects With Chronic Hepatitis C
Phase 1
Clinical Trials (1)
NCT06529796Evaluation of the Pharmacokinetics and Safety of Inaxaplin in Participants With Mild or Moderate Hepatic Impairment
Phase 1
Phase 1
Clinical Trials (1)
NCT01216046Drug-Drug Interaction Study of VX-770 and VX-809 in Healthy Subjects
Phase 1
VX-993
Pain
Phase 1
Clinical Trials (1)
NCT06226454A Phase 1 Study Evaluating the Safety, Pharmacokinetics, and Pharmacodynamics, of VX-993
Phase 1
Phase 1
Clinical Trials (1)
NCT00251199VX-950 and Peginterferon for Hepatitis C
Phase 1
VX-993
Pain
Phase 1
Clinical Trials (1)
NCT06523595Evaluation of the Pharmacokinetic Drug-drug Interactions Between VX-993 and Metformin in Healthy Adults
Phase 1
lumacaftor
Cystic Fibrosis
Phase 1
Clinical Trials (1)
NCT01899105A Phase 1 Study to Investigate the Food Effect of Lumacaftor in Combination With Ivacaftor
Phase 1
Clinical Trials (1)
NCT05955872A Study Evaluating the Relative Bioavailability and Food Effect of a Tablet Formulation of VX-147
Phase 1
VX-993
Pain
Phase 1
Clinical Trials (1)
NCT06508762A Study to Evaluate the Relative Bioavailability and Food Effect of a New Tablet Formulation of VX-993
Phase 1
PegInterferon
Chronic Hepatitis C
Phase 1
Clinical Trials (1)
NCT01704521Viral Kinetics in HCV Clearance in Subjects With Hemophilia
Phase 1
Phase 1
Clinical Trials (1)
NCT07283770Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants
Phase 1
Placebo
Pain
Phase 1
Clinical Trials (1)
NCT05418712A Microneurography (MNG) Study of VX-150 in Healthy Participants
Phase 1
telaprevir
Hepatitis C
Phase 1
Clinical Trials (1)
NCT01038167A Study to Examine the Effects of Telaprevir on the Pharmacokinetics of Cyclosporine and Tacrolimus in Healthy Adults
Phase 1
Phase 1
Clinical Trials (1)
NCT06312787A Study to Evaluate the Relative Bioavailability and Food Effect of a VX-118 Tablet Formulation
Phase 1
VX-121/TEZ/D-IVA
Cystic Fibrosis
Phase 1
Clinical Trials (1)
NCT05535959A Study to Evaluate the Relative Bioavailability of a Fixed-dose Combination Tablet of VX-121/Tezacaftor/Deutivacaftor
Phase 1
VX-548
Pain
Phase 1
Clinical Trials (1)
NCT05560464Evaluation of the Pharmacokinetics and Safety of VX-548 in Participants With Mild or Moderate Hepatic Impairment
Phase 1
VX-973
Pain
Phase 1
Clinical Trials (1)
NCT06615570A Phase 1 Dose Escalation Study of VX-973 in Healthy Participants
Phase 1

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Interview Prep Quick Facts
Portfolio: 10 approved products, 150 clinical trials
Top TAs: Rare Diseases, Infectious Diseases, Nephrology
H-1B (2023): 1 approval
Publications: 25 in PubMed
SEC Filings: 2 available
Open Roles: 295 active jobs
Portfolio Health
Growth3 (30%)
Peak6 (60%)
LOE Approaching1 (10%)
10 total products
Therapeutic Area Focus
Rare Diseases
8 marketed139 pipeline
Nephrology
14 pipeline
Respiratory
5 marketed3 pipeline
Neurology
8 pipeline
Immunology
8 pipeline
Oncology
4 pipeline
Marketed
Pipeline

Financials (FY2025)

Revenue
$9.9B11%
R&D Spend
$3.2B(32%)25%
Net Income
$3.6B
Cash
$10.5B

Hiring Trend

Actively Hiring
295
Open Roles
+297
Added
-0
Filled/Removed

Based on last 3 crawl cycles

Visa Sponsorship

Sponsors Work Visas
H-1B Petitions (FY2023)
1
Approved
1
Denied
50%
Rate

Source: USCIS H-1B Employer Data Hub