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Citi sees AstraZeneca's rare disease drug efzimfotase alfa hitting $4.4bn peak sales with broad US adult approval.

Analyst Insights
12 Oct 2026
Proactive Investors
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Bullish
Citi sees AstraZeneca's rare disease drug efzimfotase alfa hitting $4.4bn peak sales with broad US adult approval.

Citi maintains a 'buy' rating on AstraZeneca, highlighting new Phase III trial data for efzimfotase alfa, a treatment for hypophosphatasia, a rare bone disorder. Although the trial missed its primary endpoint due to a placebo effect, the drug showed significant benefits in secondary endpoints like physical function and pain reduction, especially in pediatric patients. Safety was better than the current standard treatment. AstraZeneca expects regulatory approval by mid-2027, and Citi projects peak sales could reach $4.4 billion if broad adult approval is granted, nearly doubling previous estimates.

AstraZeneca shares are trading at USD 158.19 on Pluang, down 0.63% as of Oct 12, 2026 22:31 WIB. The stock's market cap stands at $246.15 billion, with a dividend yield of 2.01%.

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