Vertex Pharmaceuticals
Rare disease drugmaker · based in Boston, United States · founded 1989 · ticker VRTX · vrtx.com
Vertex Pharmaceuticals works on 12 rare diseases we track, including Acromegaly, Congenital Adrenal Hyperplasia and 10 more. It has 19 active clinical trials, and 8 are looking for volunteers now. It also has 7 FDA-approved rare disease drugs.
The FDA is due to decide on Povetacicept for IgA nephropathy by November 30, 2026.
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Vertex Pharmaceuticals is the company that took cystic fibrosis from a disease that often killed patients before they reached adulthood and turned it into a chronic illness most patients will live decades longer with. Founded in 1989 and headquartered in Boston, Vertex trades on the NASDAQ under the ticker VRTX.
Vertex Pharmaceuticals pipeline
Each row lists the diseases Vertex Pharmaceuticals is studying at that stage, from the ones closest to approval down to the earliest work.
Povetacicept for IgA nephropathy, with a decision due by November 30, 2026.
Thalassemia 2 trials recruiting · Membranous Nephropathy recruiting · Cystic Fibrosis 4 trials · Sickle Cell Disease
Cystic Fibrosis 3 trials recruiting · Polycystic Kidney Disease 2 trials recruiting · Narcolepsy Type 1 recruiting
Myotonic Dystrophy recruiting · Polycystic Kidney Disease
Vertex Pharmaceuticals clinical trials
These are Vertex Pharmaceuticals trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
8 trials looking for volunteers now
Evaluation of Efficacy, Safety, and Tolerability of Povetacicept in Participants With Primary Membranous Nephropathy (pMN)
Tests Povetacicept, Tacrolimus
For Primary Membranous Nephropathy
Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose
Tests 14C-VX-407
For Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Evaluation of VX-581 in Healthy Participants and in Participants With Cystic Fibrosis
Tests VX-581, Placebo, D-IVA, TEZ, ELX/TEZ/IVA, VX-581, IVA
For Cystic Fibrosis
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
For DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 and Myotonic Dystrophy Type-1
Effects of VX-407 on the Pharmacokinetics of Combined Oral Contraceptives
Tests VX-407, LNG/EE, NGM/EE, NET/EE, DRSP/EE
For Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants
Tests VX-272, Placebo, Tezacaftor, Deutivacaftor
For Cystic Fibrosis
A Phase 1, First-in-human Study of VX-433
Tests VX-433, Placebo, Midazolam, Bupropion
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Tests CTX001
For Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn and Hemoglobinopathies
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FDA-approved drugs from Vertex Pharmaceuticals
From the openFDA database. Tap a drug for side effects, dosing and help paying.
Alyftrek vanzacaftor, tezacaftor, and deutivacaftor
For Cystic Fibrosis. Approved Dec 20, 2024. Taken by mouth.
Journavx suzetrigine
Approved Jan 30, 2025. Taken by mouth.
Kalydeco ivacaftor
For Cystic Fibrosis. Approved Jan 31, 2012. Taken by mouth.
Orkambi lumacaftor and ivacaftor
For Cystic Fibrosis. Approved Jul 2, 2015. Taken by mouth.
Symdeko tezacaftor and ivacaftor
For Cystic Fibrosis. Approved Feb 12, 2018.
Trikafta elexacaftor, tezacaftor, and ivacaftor
For Cystic Fibrosis. Approved Oct 21, 2019.
Casgevy exagamglogene autotemcel
For Sickle Cell Disease and Thalassemia. Taken by intravenous route.
More about Vertex Pharmaceuticals
The company spent more than 20 years on the underlying CFTR-modulator chemistry before its first approved cystic fibrosis drug, and the resulting franchise is the foundation of everything else it does today.
The current cornerstone CF drug is Trikafta, a triple combination of CFTR modulators that addresses the most common CFTR mutation, which is present in roughly 90% of CF patients. Trikafta has dramatically improved lung function, weight gain, and survival outlook for the patients eligible for it.
In December 2024, the FDA approved Alyftrek, the next-generation CF therapy combining vanzacaftor, tezacaftor, and deutivacaftor in a once-daily pill that further extends the patient population covered. Cystic fibrosis is caused by inherited mutations in the CFTR chloride channel, which lead to thick mucus buildup in the lungs and digestive tract. The Vertex modulator program has been one of the more transformative arcs in modern medicine.
Beyond CF, Vertex co-developed Casgevy (exagamglogene autotemcel) with CRISPR Therapeutics, which became the first CRISPR-based gene-editing therapy approved by the FDA. Casgevy treats sickle cell disease and transfusion-dependent beta thalassemia by editing patient cells outside the body to reactivate fetal hemoglobin, which restores healthy red blood cell function. On July 1, 2026 the FDA extended Casgevy to people aged 2 and older.
Keep reading about Vertex Pharmaceuticals
It had been approved from age 12. Casgevy represented a watershed moment for the field of gene editing as a clinical reality.
In 2025, Vertex broadened beyond rare genetics with the FDA approval of Journavx (suzetrigine) in January, the first novel non-opioid acute pain medicine in over two decades.
In 2024 Vertex also acquired Alpine Immune Sciences for $4.9 billion, gaining povetacicept, a dual BAFF/APRIL antagonist that met its primary endpoint at a planned Week 36 interim analysis of the Phase 3 RAINIER trial for IgA nephropathy, with a 49.8% placebo-adjusted reduction in urine protein-to-creatinine ratio.
Vertex completed its rolling BLA submission for povetacicept in March 2026, and the FDA accepted it for accelerated approval review with a decision date of November 30, 2026. The pipeline also includes inaxaplin (VX-147) for APOL1-mediated kidney disease, which mostly affects people of African ancestry, in the Phase 2/3 AMPLITUDE trial, and stem-cell-derived islet cell therapies for type 1 diabetes.
Vertex said on November 2, 2026 that it will focus on VX-017, a newer islet cell therapy it says could reach more patients than zimislecel, and that it expects VX-017 to enter pivotal development in 2027. On September 1, 2026, Vertex completed its approximately $10 billion acquisition of Crinetics Pharmaceuticals, adding the marketed oral acromegaly drug Palsonify (paltusotine) and moving Vertex into rare endocrine disease.
The deal also brought in atumelnant, in Phase 3 for congenital adrenal hyperplasia.
Where Vertex Pharmaceuticals trials take place
The trials above run at 620 study sites in 30 countries. Tap a country to see its states, cities and hospitals.
Rare diseases Vertex Pharmaceuticals works on
Each disease page lists every open trial, not only Vertex Pharmaceuticals's, plus approved treatments.
Acromegaly is a rare hormonal disorder caused by excessive growth hormone production, typically from a pituitary adenoma, resulting in abnormal growth of hands, feet, and facial features.
How common: About 50 to 70 people per million, according to NORD, with 3 to 11 new cases per million people each year
Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia comprises rare autosomal recessive disorders of cortisol synthesis.
How common: Classic form about 1 in 15,000 newborns (MedlinePlus Genetics); the milder non-classic form is more common
Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport.
How common: Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians
Focal Segmental Glomerulosclerosis
Focal Segmental Glomerulosclerosis (FSGS) is a kidney disease characterized by scarring of some glomeruli, the tiny filtering units that remove waste from the blood.
How common: Approximately 7% of all kidney biopsies in the United States show FSGS; incidence is 0.2-1.7 cases per 100,000 person-years
IgA nephropathy is the most common primary glomerulonephritis worldwide, caused by abnormal IgA1 antibodies depositing in the kidney's mesangium, activating complement pathways and triggering...
How common: About 2 new cases per 100,000 people each year in the U.S., and an estimated 199,000 to 208,000 Americans were living with IgA nephropathy in 2021 (Otsuka-funded study, Clinical Nephrology, 2025)
Membranous nephropathy is a rare autoimmune kidney disease where the body's own antibodies attack proteins in the kidney's filtering units (glomeruli).
How common: About 12 new cases per million people each year in the U.S.; roughly 40,000-50,000 Americans living with the disease
Myasthenia gravis is a chronic autoimmune neuromuscular disorder causing muscle weakness and rapid fatigue.
How common: 1 in 5,000 people
Myotonic Dystrophy (DM) is the most common muscular dystrophy in adults, characterized by progressive muscle weakness and myotonia—the inability of muscles to relax after contraction.
How common: Approximately 1 in 3,000 to 1 in 8,000 people globally; higher prevalence in some populations
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Vertex Pharmaceuticals's own site is vrtx.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- NIH GARD - Congenital Adrenal HyperplasiaCongenital Adrenal Hyperplasia and Cystic Fibrosis and 3 more
- National Kidney FoundationIgA Nephropathy and Membranous Nephropathy
- Acromegaly Community Inc.Acromegaly
- National Organization for Rare Disorders (NORD)Acromegaly
- CARES Foundation - Congenital Adrenal HyperplasiaCongenital Adrenal Hyperplasia
- Cystic Fibrosis FoundationCystic Fibrosis
- Travere Therapeutics — Filspari FSGS ApprovalFocal Segmental Glomerulosclerosis
- National Kidney FoundationFocal Segmental Glomerulosclerosis