Positive Ascendis data threatens BioMarin's lead in achondroplasia

18 September 2024

Ascendis Pharma (Nasdaq: ASND) has announced positive top-line data from the pivotal Phase II ApproaCH trial of TransCon CNP (navepegritide).

The outcome represents a significant challenge to BioMarin Pharmaceutical (Nasdaq: BMRN), whose stock has dropped over 15% since the news.

BioMarin currently offers the only approved drug in the USA for achondroplasia, Voxzogo (vosoritide).

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