Vertex Pharmaceuticals Inc. — Sponsor Intelligence | 25 EU Trials

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25 Registered trials

14 Recruiting

9 Completed trials

16 Countries active

20 Unique IMPs

100% Results reported

Phase III

Phase II/III

Phase II

Phase I (FiH)

Phase III: 13 Phase II/III: 3 Phase II: 4 Phase I/II: 1 Phase I (FiH): 4

Oversight events:

11 temporary halts 4 corrective measures across 5 trials

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100%

9 of 9 completed trials have published summary results (EU CTR Article 37 — 12-month obligation).

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Top indications

Cystic fibrosis 9 Sickle cell anaemia 5 Myotonic dystrophy 2 Diabetes mellitus 2 Myasthenia gravis 1

Top IMPs

EXAGAMGLOGENE AUTOTEMCEL 7 IVACAFTOR 7 TEZACAFTOR, ELEXACAFTOR, IVACAFTOR 6 TEZACAFTOR, DEUTIVACAFTOR, VANZACAFTOR 4 POVETACICEPT 2 VX-670 2 BUSULFAN 1 DEUTIVACAFTOR 1 INAXAPLIN 1 PLERIXAFOR 1 TEZACAFTOR 1 VX-264 1

All trials

25 total

A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects with Severe Sickle Cell Disease, βS/βC Genotype Pending

2023-503247-34-00 Phase III 2 countries BUSULFAN; EXAGAMGLOGENE AUTOTEMCEL; PLERIXAFOR
Safety and Efficacy of VX-828/Deutivacaftor Combination Therapy With and Without Tezacaftor in Subjects With Cystic Fibrosis Pending

2025-523400-72-00 Phase II 11 countries DEUTIVACAFTOR; IVACAFTOR; TEZACAFTOR; TEZACAFTOR, DEUTIVACAFTOR, VANZACAFTOR; TE…
A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis Recruiting

2025-523950-15-01 Phase II 1 country POVETACICEPT
Phase 2a Study of VX-407 in Subjects with ADPKD Who Have a Subset of PKD1 Gene Variants Recruiting

2024-517393-13-00 Phase II 6 countries VX-407
Evaluation of Efficacy, Safety, and Tolerability of Povetacicept in Subjects With Primary Membranous Nephropathy Recruiting

2025-521661-27-00 Phase II/III 7 countries POVETACICEPT
A Study of the Long-Term Safety and Efficacy of VX-670 in Patients With Myotonic Dystrophy Type I Recruiting

2024-517983-47-00 Phase I/II 6 countries VX-670
Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropathy Recruiting

2024-514689-38-01 Phase II 3 countries VX-993
A Phase 1/2 Study of VX-670 in Adult Subjects with Myotonic Dystrophy Type 1 Recruiting

2023-506028-10-00 Phase I (FiH) 6 countries VX-670
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Subjects 1 Year of Age and Older Recruiting

2022-503081-74-00 Phase III 4 countries TEZACAFTOR, DEUTIVACAFTOR, VANZACAFTOR
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 Impaire… Recruiting

2024-513929-23-00 Phase II/III 5 countries ZIMISLECEL
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis Subjects 1 Through 11 Years of Age Recruiting

2024-513754-29-00 Phase III 4 countries IVACAFTOR; TEZACAFTOR, DEUTIVACAFTOR, VANZACAFTOR; TEZACAFTOR, ELEXACAFTOR, IVAC…
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Prog… Recruiting

2024-514641-12-00 Phase III 2 countries EXAGAMGLOGENE AUTOTEMCEL
Phase 2/3 Adaptive Study of VX-147 in Adults and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease Recruiting

2024-515633-15-00 Phase II/III 2 countries INAXAPLIN
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease Recruiting

2024-513978-22-00 Phase III 2 countries EXAGAMGLOGENE AUTOTEMCEL
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemi… Recruiting

2024-513349-35-00 Phase III 2 countries EXAGAMGLOGENE AUTOTEMCEL
A Long-term Follow-up Study of Subjects with β-Thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic… Recruiting

2024-512654-19-00 Phase III 3 countries EXAGAMGLOGENE AUTOTEMCEL
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Subjects 12 Months of Age and Older Ended

2023-509563-24-00 Phase III 3 countries IVACAFTOR; TEZACAFTOR, ELEXACAFTOR, IVACAFTOR
A Safety, Tolerability, and Efficacy Study of VX-264 in Subjects With Type 1 Diabetes Ended

2024-515583-32-00 Phase I (FiH) 3 countries VX-264
A Phase 3 Study of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Ended

2024-514173-22-00 Phase III 16 countries TEZACAFTOR, DEUTIVACAFTOR, VANZACAFTOR
Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor Long-term Safety and Efficacy in Subjects Without F508del Ended

2024-515637-14-00 Phase III 13 countries IVACAFTOR; TEZACAFTOR, ELEXACAFTOR, IVACAFTOR
A Phase 1/2 Study of VX-522 in Subjects with Cystic Fibrosis Ended

2023-504786-23-00 Phase I (FiH) 6 countries IVACAFTOR; VX-522
Evaluation of ELX/TEZ/IVA in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age Ended

2023-503230-49-00 Phase III 3 countries IVACAFTOR; TEZACAFTOR, ELEXACAFTOR, IVACAFTOR
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Subjects 2 Years and Older Ended

2024-515606-90-00 Phase III 1 country IVACAFTOR; TEZACAFTOR, ELEXACAFTOR, IVACAFTOR
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 a… Ended

2024-516067-83-00 Phase I (FiH) 2 countries EXAGAMGLOGENE AUTOTEMCEL

Showing 1–24 of 25 trials

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