Vertex Reports Q1 2025 Results; Raises Revenue Outlook Amid Product Launch Momentum

Vertex Pharmaceuticals posted Q1 2025 revenue of $2.77 billion, up 3% year over year, driven by growth in cystic fibrosis (CF) therapies and early contributions from new product launches ALYFTREK and JOURNAVX. U.S. revenue rose 9% to $1.66 billion, while international sales declined 5%, impacted by IP issues in Russia.
The company raised the lower end of its 2025 revenue guidance from $11.75 billion to $11.85 billion, now expecting between $11.85 billion and $12.0 billion. Net income was $646 million on a GAAP basis, down from $1.1 billion in Q1 2024, due largely to a $379 million impairment charge tied to the discontinued VX-264 diabetes program. Non-GAAP net income was $1.05 billion.
Key updates:
• ALYFTREK, the next-gen CF modulator, is now approved in the U.S. and U.K., with EU approval expected in H2 2025.
• CASGEVY, a CRISPR-based therapy for sickle cell disease and beta thalassemia, has over 65 global treatment centers activated and ~90 patients collected to date.
• JOURNAVX, a non-opioid acute pain therapy approved in January, has filled over 20,000 prescriptions since March and has broad commercial coverage.
• Multiple pivotal trials continue, including povetacicept for kidney diseases (IgAN and pMN) and zimislecel for type 1 diabetes, with filings targeted for 2026.
• Vertex has $11.4 billion in cash and marketable securities and continues to invest heavily in R&D.

Vertex Pharmaceuticals announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of ALYFTREK (deutivacaftor/tezacaftor/vanzacaftor) for people with cystic fibrosis aged six and older who have at least one non-class I mutation in the CFTR gene. Clinical trials showed that ALYFTREK was non-inferior to KAFTRIO in improving lung function (ppFEV1) and superior at lowering sweat chloride levels, a key indicator of disease severity. ALYFTREK is already licensed in the U.S. and UK and is under review in several other countries. If approved, the therapy could expand treatment eligibility to more patients by achieving closer-to-normal sweat chloride levels.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Paul Negulescu, Ph.D. Senior Vice President, Vertex has been awarded the 2025 Canada Gairdner International Award “for pioneering research into the cellular and molecular mechanisms underlying the genetic disease cystic fibrosis, leading to the development of transformative drug therapies based on these mechanisms, thereby improving and saving countless lives.” Negulescu shares the award with Michael J. Welsh, M.D., University of Iowa.

European Commission Expands Approval for Vertex’s KAFTRIO® to Treat More Children With Cystic Fibrosis

Vertex Pharmaceuticals announced that the European Commission has approved an expanded label for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) combined with ivacaftor. The approval now includes all patients aged two years and older with at least one non-class I CFTR gene mutation. This expansion means approximately 4,000 additional patients in the EU are now eligible for a treatment that targets the underlying cause of their cystic fibrosis (CF) for the first time.

KAFTRIO, already used by over 68,000 people across more than 60 countries, is designed to restore CFTR protein function and improve outcomes such as lung function and quality of life. Vertex confirmed that countries with existing reimbursement agreements, such as Germany, Sweden, and Austria, will soon offer access to the expanded indication. The company is also working to broaden access across the EU.

Cystic fibrosis is a rare genetic disease that affects multiple organs and is often fatal. The expanded approval represents a significant step forward in reaching underserved patients with rare CF mutations.

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its first quarter 2025 financial results on Monday, May 5, 2025 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET. To access the call, please dial (833) 630-2124 (U.S.) or +1 (412) 317-0651 (International) and reference the “Vertex Pharmaceuticals First Quarter 2025 Earnings Call.”

EU Expands Approval of Vertex’s KAFTRIO to Treat More Young Cystic Fibrosis Patients

Vertex Pharmaceuticals announced that the European Commission has approved an expanded label for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor. The treatment is now authorized for cystic fibrosis patients aged 2 and older with at least one non-class I CFTR gene mutation. This expansion means around 4,000 additional individuals in the EU are now eligible for a therapy that targets the underlying cause of the disease.

Existing reimbursement agreements in countries like Austria, Denmark, and Sweden will facilitate swift access, and Vertex plans to work with other EU health authorities to broaden availability. KAFTRIO helps improve CFTR protein function, enhancing the flow of salt and water in affected organs and ultimately easing symptoms such as thick mucus and lung infections. Vertex’s CF treatments now serve over 68,000 patients globally, two-thirds of those eligible for CFTR modulators.

Vertex Pharmaceuticals Reports $400 Million Impairment Charge Following VX-264 Program Update

Vertex Pharmaceuticals announced it will discontinue further development of its type 1 diabetes candidate VX-264 after results from a Phase 1/2 trial. Following this decision, Vertex began evaluating the intangible assets associated with the program and expects to record a non-cash impairment charge of approximately $400 million for the first quarter and full year of 2025.

Details of the impairment will be included in Vertex’s upcoming Quarterly Report on Form 10-Q for the quarter ending March 31, 2025.

Vertex noted that this announcement contains forward-looking statements subject to risks and uncertainties, particularly regarding the expected financial impact and accounting treatment. These forward-looking statements are based on current information and are subject to change.

**Vertex announces Type 1 diabetes program update: VX-264 ends, zimislecel progresses toward regulatory submissions**

On March 28, 2025, Vertex Pharmaceuticals announced key updates to its type 1 diabetes (T1D) portfolio. The company has completed enrollment and dosing for Parts A and B of the Phase 1/2 study of VX-264, a cell therapy encapsulated in an immunoprotective device. While VX-264 was generally safe and well tolerated, it failed to show efficacy in increasing insulin production, measured by C-peptide levels. As a result, Vertex will not advance VX-264 to further clinical development but will continue further analyses, including of explanted devices.

Meanwhile, the pivotal trial of zimislecel (VX-880), a fully differentiated islet cell therapy with standard immunosuppression, is on track to complete enrollment and dosing in the first half of 2025. Regulatory submissions are expected in 2026. Zimislecel has received multiple regulatory designations, including RMAT and Fast Track in the US and PRIME status in Europe. Vertex anticipates an initial approval could benefit about 60,000 people with severe T1D in the US and Europe.

Vertex also reaffirmed its commitment to developing additional research-stage approaches for T1D, including gene-edited hypoimmune islet cell therapies and novel encapsulation technologies to reduce or eliminate the need for standard immunosuppression.

Carmen Bozic, M.D., Chief Medical Officer, stated, “Today’s data show that more work needs to be done to advance the ‘cells plus device’ program, and we are committed to doing so. We’re also very pleased with the rapid progress of our zimislecel program.”

More information can be found at [www.vrtx.com](https://www.vrtx.com) or via BusinessWire [here](https://www.businesswire.com/news/home/20250328622122/en/).

Vertex Pharmaceuticals announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), a once-daily cystic fibrosis treatment for patients aged 6 and older with at least one responsive mutation in the CFTR gene. The approval expands access to additional mutations not covered by previous CFTR modulator therapies.

In clinical trials, Alyftrek demonstrated non-inferiority in lung function (ppFEV1) and further reduced sweat chloride levels compared to the existing combination therapy of ivacaftor/tezacaftor/elexacaftor with ivacaftor. Researchers hope these improvements will lead to a lower risk of CF-related complications over time.

Vertex is working with the UK’s National Institute for Health and Care Excellence (NICE) and the NHS to make the therapy available to eligible patients as soon as possible. The drug was approved by the U.S. FDA in December 2024, and regulatory reviews are ongoing in Europe, Canada, Switzerland, Australia, and New Zealand.

Vertex continues to be a leader in CF treatment, with over 68,000 patients currently using its therapies across more than 60 countries. The company remains focused on advancing its pipeline in CF and other serious diseases.

Vertex Pharmaceuticals announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for expanding the label of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor to include people with cystic fibrosis (CF) aged 2 years and older with at least one non-class I CFTR mutation. If approved, around 4,000 additional CF patients in the European Union will have access to treatment targeting the disease’s underlying cause for the first time.

KAFTRIO is currently approved in the EU for patients with at least one copy of the F508del mutation in the CFTR gene. The therapy improves CFTR protein function, helping clear thick mucus from the lungs and improving lung function. Vertex’s CF medicines currently treat over 68,000 patients across more than 60 countries.

Cystic fibrosis is a progressive, life-shortening genetic disease affecting multiple organs. The median age of death is in the 30s, but treatment advances continue to improve projected survival. The CHMP's recommendation moves Vertex closer to its goal of expanding CF treatment access to all eligible patients.

The European Commission is expected to make a final decision on the label expansion in the coming months.

Vertex Pharmaceuticals has secured a reimbursement agreement with NHS England, granting eligible sickle cell disease (SCD) patients access to *CASGEVY® (exagamglogene autotemcel)*, a CRISPR/Cas9 gene-edited therapy. The agreement follows the National Institute for Health and Care Excellence's (NICE) positive guidance and builds on a prior agreement for transfusion-dependent beta thalassemia (TDT) patients. Vertex is also working with hospitals across England to establish authorized treatment centers. This milestone marks a significant advancement in treatment options for SCD patients, offering a potential one-time therapy to address the disease’s underlying cause. (*Vertex Pharmaceuticals*)

Vertex Pharmaceuticals has announced FDA approval for *JOURNAVX™ (suzetrigine)*, the first non-opioid oral pain signal inhibitor and the first new class of pain medication in over 20 years. Designed for adults experiencing moderate-to-severe acute pain, JOURNAVX offers an effective, well-tolerated alternative without the addictive potential of opioids. With over 80 million Americans prescribed pain medication annually—40 million of whom receive opioids—this approval marks a significant shift in acute pain management. Vertex has set the wholesale acquisition cost at $15.50 per 50mg pill and plans to expand clinical trials evaluating suzetrigine for neuropathic pain. (*Vertex Pharmaceuticals*)

Vertex Pharmaceuticals and Zai Lab have entered into an exclusive agreement to develop and commercialize povetacicept in mainland China, Hong Kong SAR, Macau SAR, Taiwan, and Singapore. Povetacicept, a dual antagonist targeting BAFF and APRIL cytokines, is being studied for B cell-mediated diseases, including IgA nephropathy. Under the partnership, Zai Lab will advance clinical trials, manage regulatory submissions, and oversee commercialization upon approval. Vertex will receive upfront payments, regulatory milestones, and tiered royalties. This collaboration aims to accelerate access to povetacicept for patients in the licensed regions. (Source: Vertex Pharmaceuticals)