2025-06-03-Jefferies-Ascendis_Pharma（ASND）_Navepegritide的优先审评重申12个月数据可能足以获批_9页_331kb

报告摘要

Navepegritide's NDA for treating children with achondroplasia accepted under FDA priority review with 11/30 PDUFA date.
Pivotal Phase 3 data show +1.49 cm/year LS mean difference vs placebo in 2-11yo subjects, comparable to Voxzogo, supporting 12-month data for approval.
Buy rating with $229 price target; market cap is $10.5B.
Ph2 COACH combo trial with Skytrofa may enhance adoption if additive benefits are observed.
Key risks include clinical/regulatory uncertainties; competition with Voxzogo.
Exhibit comparisons highlight efficacy data against competitor drugs.

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