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    Is CASGEVY’s Pediatric Breakthrough And Priority Review Altering The Investment Case For Vertex (VRTX)?

    Simply Wall St·12/20/2025 18:15:16
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    • Vertex Pharmaceuticals recently reported new clinical data showing that all evaluable children aged 5–11 treated with CASGEVY achieved key endpoints in sickle cell disease and transfusion-dependent beta thalassemia, and also secured an FDA National Priority Voucher to accelerate review in this younger age group.
    • By pairing strong pediatric efficacy signals with an expedited U.S. regulatory review pathway, Vertex is strengthening CASGEVY’s potential role in reshaping treatment for severe inherited blood disorders.
    • We’ll now explore how CASGEVY’s promising pediatric results and accelerated FDA review could influence Vertex’s broader investment narrative.

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    Vertex Pharmaceuticals Investment Narrative Recap

    To own Vertex, you need to believe the company can gradually reduce its dependence on cystic fibrosis drugs by building durable franchises in gene editing, pain, and kidney disease. The CASGEVY pediatric data and FDA National Priority Voucher support one of the key near term catalysts: broadening access for this gene edited therapy. At the same time, heavy R&D spend and uncertain outcomes across the broader pipeline remain the most important execution risk.

    Among recent developments, Vertex’s raised 2025 revenue guidance to US$11.9–12.0 billion stands out, reflecting uptake of CASGEVY alongside the CF portfolio, ALYFTREK, and JOURNAVX. In that context, positive CASGEVY data in 5–11 year olds, plus an accelerated U.S. review pathway, tie directly into one of the main growth drivers analysts are watching: how quickly Vertex can turn its gene editing advances into a meaningful, diversified revenue stream.

    Yet while CASGEVY could ease dependence on cystic fibrosis, investors should also be aware that...

    Read the full narrative on Vertex Pharmaceuticals (it's free!)

    Vertex Pharmaceuticals' narrative projects $14.9 billion revenue and $5.6 billion earnings by 2028. This requires 9.4% yearly revenue growth and about a $2.0 billion earnings increase from $3.6 billion today.

    Uncover how Vertex Pharmaceuticals' forecasts yield a $485.36 fair value, a 6% upside to its current price.

    Exploring Other Perspectives

    VRTX 1-Year Stock Price Chart
    VRTX 1-Year Stock Price Chart

    Some of the most optimistic analysts already expected Vertex to reach about US$16.9 billion in revenue and US$7.8 billion in earnings by 2028, and they see CASGEVY’s gene editing progress as a key reason Vertex could capture an outsized share of high margin, transformative therapies compared with consensus expectations.

    Explore 8 other fair value estimates on Vertex Pharmaceuticals - why the stock might be worth 25% less than the current price!

    Build Your Own Vertex Pharmaceuticals Narrative

    Disagree with existing narratives? Create your own in under 3 minutes - extraordinary investment returns rarely come from following the herd.

    • A great starting point for your Vertex Pharmaceuticals research is our analysis highlighting 3 key rewards that could impact your investment decision.
    • Our free Vertex Pharmaceuticals research report provides a comprehensive fundamental analysis summarized in a single visual - the Snowflake - making it easy to evaluate Vertex Pharmaceuticals' overall financial health at a glance.

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    This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.