Vertex Pharmaceuticals Inc. (VRTX)

Vertex is a biotechnology company making medicines for serious diseases, with a focus on specialty markets. It has approved medicines in four places: cystic fibrosis, sickle cell disease, transfusion dependent beta thalassemia, and acute pain. At the end of 2025 it had approximately 6,400 employees, approximately 5,200 of them based in the United States and approximately 1,200 outside it.

Cystic fibrosis

Cystic fibrosis is a life-shortening genetic disease caused by a defective or missing CFTR protein, the result of mutations in the CFTR gene. Without working CFTR protein, salt and water flow poorly into and out of cells in several organs, including the lungs, where mucus builds up and causes chronic infection and progressive damage. The company's CFTR modulators, ivacaftor, deutivacaftor, lumacaftor, tezacaftor, elexacaftor and vanzacaftor, work on the underlying cause by improving how the CFTR protein functions.

Five medicines are on the market: ALYFTREK, TRIKAFTA, sold as KAFTRIO in some regions, SYMDEKO, sold as SYMKEVI, ORKAMBI and KALYDECO. The company says they are being used by nearly three quarters of the approximately 97,000 people with cystic fibrosis in the United States, Europe, Australia and Canada, and estimates approximately 112,000 people with the disease across all its target markets. The medicines are reimbursed or accessible in more than 60 countries across six continents. ALYFTREK is the most recently approved triple combination, taken once daily, which the company reports was non-inferior to TRIKAFTA on a measure of lung function.

Sickle cell disease and beta thalassemia

Both are inherited blood disorders arising from gene mutations that alter hemoglobin, the protein in red blood cells that carries oxygen. In sickle cell disease a single amino acid change makes red cells change shape when oxygen is low; the sickled cells block blood flow, causing severe pain, organ damage and a shortened life. In transfusion dependent beta thalassemia, loss-of-function mutations in the same gene cause severe anemia, and patients need regular blood transfusions as often as every two to four weeks, which eventually builds up iron in the body and damages organs.

CASGEVY is an ex-vivo, non-viral CRISPR/Cas9 gene-editing therapy for the severe forms of both, approved in the United States, Europe, Canada and the Middle East. To receive it, a patient first undergoes a treatment at an authorised treatment centre that mobilises hematopoietic stem and progenitor cells from the bone marrow into the bloodstream; those cells are collected and sent to a manufacturing facility, where they are isolated and the gene edit is made in a non-coding intron of the BCL11A gene; the edited cells go back to the treatment centre; the patient is preconditioned with a myeloablative treatment that clears the bone marrow to make space; and the edited cells are infused and engraft, after which fetal hemoglobin levels rise. The company estimates approximately 60,000 people with severe forms of the two diseases are or could become eligible in the geographies where it is approved. During 2025 approximately 300 people started treatment, 147 had their first cell collection and 64 received infusions.

Acute pain

JOURNAVX, suzetrigine, is described in the filing as a first-in-class oral inhibitor of pain signals, highly selective for the voltage-gated sodium channel NaV1.8, which it says relieves pain without the addictive potential of opioids. The Food and Drug Administration approved it in January 2025 for moderate-to-severe acute pain in adults. It reached United States pharmacies in March 2025, and by the end of that year more than 550,000 prescriptions had been written and filled across hospital and retail settings. Access is secured with all three national pharmacy benefit managers, and as of January 2026 more than 200 million individuals across commercial and government payors had coverage, which the company puts at two-thirds of United States covered lives, with 21 states covering it through Medicaid.

What is coming, and how the medicines are sold

Five pivotal programmes are in progress, among them povetacicept, a dual inhibitor of B cell activating factor, for IgA nephropathy. The wider mid- and late-stage pipeline covers APOL1-mediated kidney disease, neuropathic pain, type 1 diabetes, primary membranous nephropathy, autosomal dominant polycystic kidney disease and myotonic dystrophy type 1.

The medicines are sold primarily to a limited number of specialty pharmacies and specialty distributors globally, and to certain major wholesalers in the United States, who resell to patients, healthcare providers, retail pharmacies, hospitals or treatment centres. Outside the United States sales run mainly through distributor arrangements and to retail pharmacies, hospitals and clinics, many of them government-owned or supported. In certain markets CASGEVY is sold directly to authorised treatment centres instead. The company contracts with government agencies so its medicines are eligible for purchase or reimbursement, and promotes directly to healthcare professionals and organisations.

From Vertex Pharmaceuticals' annual report on Form 10-K for the year ended 31 December 2025. The filing