Vertex PharmaceuticalsActive
Once-daily next-generation CFTR modulator (vanzacaftor/tezacaftor/deutivacaftor). The Oct. 9, 2026 company release says the US indication is patients 6 years and older with a responsive CFTR variant. Use in children ages 2 to 5 is investigational.
Latest update Oct 31, 2026
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At the 2026 North American Cystic Fibrosis Conference, Vertex reported interim open-label data from Study VX22-121-106 Cohort 2 in children ages 2 to 5. After at least 48 weeks, mean fecal elastase-1 was 239.7 mcg/g, a mean increase of 110.2 mcg/g, and 48.6% reached at least 200 mcg/g. Of 48 children eligible for a pancreatic-enzyme discontinuation substudy, 18 (37.5%) had stopped for a mean of 8.6 weeks. Across 66 enrolled children, 50% were not on pancreatic enzyme therapy at the data cut. Those ages remain investigational in the US.
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Announced next steps from Vertex Pharmaceuticals that mention Alyftrek (vanzacaftor/tezacaftor/deutivacaftor).
2026 ongoing
Convert Trikafta patients onto the once-daily Alyftrek and expand international launches to extend the cystic fibrosis franchise. Alyftrek is now approved in the US, UK, EU (covering an addressable population of about 31,000 CF patients), Canada, Switzerland, Australia and New Zealand. Vertex signed a Letter of Intent with the pan-Canadian Pharmaceutical Alliance on July 6, 2026 following positive reimbursement recommendations from CDA-AMC and INESSS, and has secured reimbursement agreements in Scotland, Spain, Sweden, Switzerland, New Zealand, Israel and Finland, with more countries in progress. Vertex also plans global regulatory submissions to extend the label to children ages 2-5.
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Vertex's flagship cystic fibrosis triple-combination CFTR modulator (elexacaftor/tezacaftor/ivacaftor), effective for roughly 90% of CF patients and the company's dominant revenue engine - now complemented by the once-daily next-generation Alyftrek.
A first-in-class oral, non-opioid pain medicine (VX-548) that selectively inhibits the NaV1.8 sodium channel to block pain signals in the peripheral nervous system before they reach the brain - the first new class of pain drug in over 20 years.
The first approved CRISPR/Cas9 gene-editing medicine (exagamglogene autotemcel), a one-time therapy for sickle cell disease and transfusion-dependent beta thalassemia, developed and commercialized with CRISPR Therapeutics.
An allogeneic, stem-cell-derived, fully differentiated islet cell therapy for type 1 diabetes designed to restore the body's own insulin production - a potential functional cure delivered as a one-time infusion.
An oral, once-daily somatostatin receptor agonist for acromegaly, acquired through the Crinetics Pharmaceuticals deal - Vertex's first marketed product outside CF, pain, and gene therapy, and the anchor of its new endocrine-disease business.
An oral pipeline candidate for endocrine disease acquired with Crinetics Pharmaceuticals, part of the endocrinology pipeline Vertex and Crinetics said could reach $5B+ in combined peak annual sales alongside Palsonify.
Dual BAFF and APRIL inhibitor being studied for IgA nephropathy. Not approved. Vertex said that if the FDA approves it, the planned launch form is a low-volume subcutaneous autoinjector given at home every 4 weeks.
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Oklo
Oklo's fast-fission powerhouse product line is designed for build-own-operate deployment, targeting customers that want firm clean power in a smaller footprint than traditional large reactors.
Oklo
Oklo is developing an advanced fuel center to recycle used nuclear fuel and fabricate metal fuel for Aurora and other fast-reactor applications.
Oklo
Oklo's isotope business combines the Idaho Radiochemistry Laboratory, a planned pilot reactor in Texas, and a future multi-reactor foundry to supply critical medical, industrial, research, defense, and space isotopes.
TerraPower
Sodium-cooled fast reactor with integrated molten salt energy storage, enabling flexible output from 345 MWe to 500 MWe peak.