Will FDA Accelerated Pathway for Delpacibart Zotadirsen Reshape Avidity Biosciences' (RNA) Rare Disease Trajectory?

• In November 2025, Avidity Biosciences announced the launch of its Managed Access Program for the investigational therapy delpacibart zotadirsen for eligible Duchenne muscular dystrophy (DMD44) patients in the United States, aligning with the FDA on a 2026 accelerated approval submission pathway.
• This move reflects significant clinical and regulatory progress, as the therapy has earned multiple expedited designations from both U.S. and European regulators.
• We'll explore how securing FDA pathway alignment for delpacibart zotadirsen could influence Avidity Biosciences' long-term position in rare disease therapeutics.

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What Is Avidity Biosciences' Investment Narrative?

To believe in Avidity Biosciences as a shareholder, you're really buying into the potential of its RNA-targeted therapies to break new ground in rare disease treatment, especially Duchenne muscular dystrophy. The recent news, launching the Managed Access Program and aligning with the FDA for an accelerated approval pathway, is a crucial short-term catalyst that could improve regulatory visibility and confidence. However, this progress comes in the context of steep operating losses and ongoing unprofitability, which have only grown in the past year. Any gains from pipeline milestones have to be weighed against significant cash burn and the risk of fundraising or dilution. Adding to the mix is the pending Novartis acquisition, which may reshape catalysts and risks ahead. For now, this latest regulatory step suggests a stronger position in the race toward commercialization, but investors should be mindful that even positive trial data does not guarantee market success or eventual profitability. Still, heavy operational losses remain a real concern that investors should be aware of.

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Exploring Other Perspectives

Among Simply Wall St Community members, intrinsic value opinions range widely, from US$30.34 up to US$74, reflecting just two distinct perspectives. Beneath this spread, most agree that regulatory milestones remain crucial in shaping eventual outcomes, especially as losses persist. This diversity speaks to the need for readers to examine several viewpoints before forming an outlook on Avidity Biosciences.

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Build Your Own Avidity Biosciences Narrative

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

• A great starting point for your Avidity Biosciences research is our analysis highlighting 1 key reward and 3 important warning signs that could impact your investment decision.
• Our free Avidity Biosciences research report provides a comprehensive fundamental analysis summarized in a single visual - the Snowflake - making it easy to evaluate Avidity Biosciences' 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.

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