Vertex Pharmaceuticals

Vertex Pharmaceuticals works on 14 rare diseases tracked on Trial Friend, including Alpha-1 Antitrypsin Deficiency, Cystic Fibrosis, Focal Epilepsy and 11 more, with 17 recruiting clinical trials and 7 FDA-approved rare disease drugs.

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. 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. 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 earlier-stage pipeline includes inaxaplin (VX-147) for APOL1-mediated kidney disease, which mostly affects people of African ancestry, and zimislecel, a stem-cell-derived islet therapy for type 1 diabetes. 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.