Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) has released a comprehensive set of business and pipeline updates ahead of its scheduled investor meetings in January, underscoring strong commercial momentum and continued advancement across its research and development portfolio. These updates will be highlighted during the company’s webcast presentation at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026, at 5:15 p.m. ET / 2:15 p.m. PT.
Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex, emphasized the company’s trajectory following a year of meaningful execution and progress. According to Dr. Kewalramani, 2025 marked a period of robust commercial performance and accelerated R&D advancement, positioning Vertex for sustained growth and multiple value-driving milestones in 2026. The company remains focused on broadening its commercial footprint across several disease areas, advancing its emerging renal portfolio—including the potential near-term launch of povetacicept—and progressing a diverse mid- and late-stage clinical pipeline. Vertex believes it is well positioned to reach significantly more patients worldwide while continuing to deliver durable shareholder value.
Established Disease Areas with Approved Medicines
Cystic Fibrosis (CF)
Vertex continues to lead in cystic fibrosis, with multiple approved and next-generation therapies. ALYFTREK® has received regulatory approval across major global markets, including the U.S., U.K., European Union, Canada, Australia, and others, for patients aged six years and older with at least one responsive CFTR mutation. Reimbursed access is already available in several countries, and efforts are ongoing to expand coverage globally. Vertex plans to present data from studies in children aged two to five years and submit regulatory filings in 2026, with a pivotal study in children aged one to two years expected to begin the same year.
TRIKAFTA® also continues to expand its label. Following positive clinical results in patients aged one to under two years, Vertex anticipates initiating global regulatory submissions in the first half of 2026. Meanwhile, next-generation CFTR modulators remain a priority, with VX-828 entering clinical studies as the first corrector in the 3.0 class and VX-581 advancing into Phase 1 development.
For patients who cannot benefit from CFTR modulators, Vertex and Moderna are developing VX-522, an mRNA-based CFTR therapy. Dosing in the multiple ascending dose portion of its Phase 1/2 study is expected to conclude in 2026, with data anticipated in the second half of the year. Reflecting expanded screening and diagnosis, Vertex has also updated its epidemiological estimates, now projecting approximately 112,000 people with CF across target markets.
Severe Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)
CASGEVY® continues to gain global traction, with approvals now spanning North America, Europe, and the Middle East for patients aged 12 years and older. In 2025, Vertex surpassed its target of $100 million in CASGEVY revenue, supported by more than 60 patient infusions. Positive pivotal data in children aged five to 11 years were presented at the 2025 ASH Annual Meeting, and regulatory submissions are expected to begin in early 2026. The FDA has granted a Commissioner’s National Priority Voucher for this pediatric submission, accelerating review timelines. Collectively, these developments are expected to drive substantial revenue growth in 2026 and beyond.
Acute Pain
JOURNAVX® (suzetrigine) represents Vertex’s first approved therapy in acute pain. Since its FDA approval in early 2025, more than half a million prescriptions have been written across hospital and retail settings. The company has secured coverage with all three major U.S. pharmacy benefit managers, providing access to more than 200 million covered lives. Vertex expects strong prescription growth in 2026 and plans to submit a regulatory application in Canada in the first half of the year.
Programs in Pivotal and Mid-Stage Development
Vertex continues to advance multiple late-stage programs. In peripheral neuropathic pain, Phase 3 enrollment for suzetrigine in diabetic peripheral neuropathy is expected to complete by the end of 2026, while VX-993 remains in Phase 2 development. Updated estimates now place the U.S. diabetic peripheral neuropathy population at approximately 2.5 million.
In kidney disease, povetacicept represents a cornerstone of Vertex’s emerging renal franchise. A rolling biologics license application for accelerated approval in IgA nephropathy is underway, supported by Breakthrough Therapy Designation and priority review. Parallel studies in primary membranous nephropathy are ongoing, with both U.S. and European regulators granting expedited development designations. Vertex has also expanded its estimates of the global patient population for these indications.
Inaxaplin for APOL1-mediated kidney disease remains on track, with interim analysis data expected in late 2026 or early 2027. In type 1 diabetes, enrollment in the zimislecel Phase 1/2/3 study is complete, though dosing has been temporarily paused pending manufacturing review. The program continues to receive strong regulatory support across multiple regions.
Mid-stage programs include VX-407 for autosomal dominant polycystic kidney disease, VX-670 for myotonic dystrophy type 1, and plans to initiate a Phase 2 study of povetacicept in generalized myasthenia gravis in 2026.
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