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    Did Strong Pediatric CASGEVY Data and FDA Priority Review Just Shift Vertex Pharmaceuticals' (VRTX) Investment Narrative?

    Simply Wall St·12/13/2025 17:20:31
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    • Vertex Pharmaceuticals recently reported clinical data showing that all evaluated children aged 5–11 with severe sickle cell disease or transfusion-dependent beta thalassemia treated with CASGEVY met key efficacy endpoints, and the FDA granted a Priority Review Voucher to accelerate review of the therapy in this younger age group.
    • This progress in extending CASGEVY’s use to pediatric patients could meaningfully broaden its addressable patient population and support longer-term utilization of Vertex’s gene-editing platform.
    • We’ll now explore how the strong pediatric CASGEVY results and Priority Review Voucher could influence Vertex’s evolving investment narrative.

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    Vertex Pharmaceuticals Investment Narrative Recap

    To own Vertex, you generally need to believe its cystic fibrosis core can fund a broader portfolio in gene editing, kidney disease, and pain while managing pricing and regulatory pressures. The strong pediatric CASGEVY data and Priority Review Voucher directly support the gene-editing story and may reinforce CASGEVY as a key near term catalyst, though they do not remove the larger risks around R&D setbacks and potential pricing pushback on high-cost therapies.

    Among recent updates, the CFO’s comments about intentionally increasing operating expenses to scale multiple launches and advance the kidney and pain portfolios are especially relevant. They highlight that management is willing to absorb higher near term costs to support launches like CASGEVY, JOURNAVX, and future kidney assets, which ties directly into how successfully Vertex can convert today’s clinical wins into a durable, more diversified revenue base.

    Yet, even as CASGEVY gains momentum, investors should be aware that growing pricing pressure on high-cost therapies could eventually...

    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 7% 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 revenue to reach about US$16.9 billion and earnings US$7.8 billion by 2028, assuming Vertex’s gene editing leadership would translate into very high margin growth. The new pediatric CASGEVY data could either reinforce that bullish view or prompt you to reassess how much future pricing and competitive risks might still weigh on those expectations.

    Explore 8 other fair value estimates on Vertex Pharmaceuticals - why the stock might be worth 24% 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.