Phases, active trials: Phase 1: 6, Phase 2: 6, Phase 3: 18, Other: 2
Vertex Pharmaceuticals has achieved a near-monopoly in cystic fibrosis (CF) through its CFTR modulator franchise. Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor) is the standard of care for approximately 90% of CF patients with at least one F508del mutation, generating over $9 billion in annual revenue. Vertex is now pursuing the remaining ~10% of CF patients with the next-generation triple combination VX-522, designed for patients whose mutations do not respond to current modulators. Beyond CF, Vertex is executing a bold expansion into adjacent rare diseases: VX-880 and CTX001/exa-cel (partnered with CRISPR Therapeutics) for sickle cell disease and beta-thalassemia, VX-147 (inaxaplin) targeting APOL1-mediated kidney disease, a condition affecting Black patients disproportionately, and suzetrigine (VX-548) for acute and neuropathic pain via selective NaV1.8 sodium channel blockade, which received FDA approval as the first new non-opioid pain mechanism in decades.
- Cystic fibrosis (CFTR modulators)
- Sickle cell disease / beta-thalassemia (gene editing)
- APOL1-mediated kidney disease (VX-147)
- Non-opioid pain (NaV1.8 inhibition)
- Alpha-1 antitrypsin deficiency (AATD)
- Type 1 diabetes (islet cell therapy)
- Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor): CF CFTR modulator
- VX-522: next-gen CFTR triple combo for non-F508del patients
- Exa-cel (exagamglogene autotemcel): CRISPR gene editing, SCD/beta-thalassemia
- VX-147 (inaxaplin): APOL1 inhibitor, kidney disease
- Suzetrigine (VX-548): NaV1.8 inhibitor, acute/neuropathic pain
- VX-880: allogeneic islet cell therapy, Type 1 diabetes
A primary completion date pushed out, enrollment cut, or a primary endpoint changed in the last 90 days. Active trials only.
| Trial | Warning signs | Status |
|---|---|---|
| A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants… NCT04786262 | Primary completion moved at least 6 months later Primary endpoint added | Recruiting |
3 more on the Trials at Risk page. See which trials →
Phase 2 and Phase 3 trials with a primary completion date in the next 180 days, as ClinicalTrials.gov estimates it today. Active trials only.
| Trial | Scheduled | Phase |
|---|---|---|
| A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination… NCT05444257 | Oct 30, 2026 · Estimated | Phase 3 |
6 more on the Completions page. See the calendar →
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Start monitoring free| Condition / indication | Active trials |
|---|---|
| Cystic Fibrosis | 8 |
| Autosomal Dominant Polycystic Kidney Disease (ADPKD) | 4 |
| Diabetic Peripheral Neuropathic Pain | 4 |
| Hemoglobinopathies | 4 |
| Beta-Thalassemia | 3 |
| Genetic Diseases, Inborn | 3 |
| Hematologic Diseases | 3 |
| Sickle Cell Disease | 3 |
Vertex's expansion beyond CF is the most-watched rare disease story in pharma. VX-147 could be the first APOL1-targeted therapy, a massive unmet need in a genetically defined population. Their islet cell therapy program (VX-880) is the leading signal for a functional T1D cure. Monitoring Vertex's trial registrations reveals expansion into ADPKD, AATD, and new pain indications.
DataLookout monitors ClinicalTrials.gov daily and surfaces changes to Vertex Pharmaceuticals's registered studies: new trial registrations, status transitions (for example "Not yet recruiting" to "Recruiting"), site additions, and protocol amendments. This is the intelligence layer that bridges the gap between quarterly investor calls and day-to-day pipeline developments.
- Sponsor normalization: Vertex Pharmaceuticals may register trials under multiple legal entity names. DataLookout normalizes all related entities to a single canonical sponsor for complete pipeline coverage.
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Get started free| Trial | Status | Latest tracked change | Seen |
|---|---|---|---|
| A Phase 3 Open-Label Study of Long-term Treatment With Povetacicept in Adults… NCT07807995 | Enrolling by Invitation | Start date changed from estimate of Sep 18, 2026 to Sep 17, 2026 | 2026-10-07 |
| Effects of VX-407 on the Pharmacokinetics of Combined Oral Contraceptives NCT07792408 | Recruiting | Recruitment opened | 2026-10-01 |
| Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in… NCT07437105 | Recruiting | Interventions modified | 2026-09-24 |
| Evaluation of Efficacy, Safety, and Tolerability of Povetacicept in… NCT07204275 | Recruiting | Trial sites expanded: 111 → 114 locations | 2026-09-03 |
| A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants… NCT04786262 | Recruiting | Interventions modified | 2026-09-02 |
Each trial page shows every change DataLookout has recorded for that trial.