Ascendis Challenges BioMarin’s Dominance with Promising Achondroplasia Drug Data

Ascendis Pharma's TransCon CNP has shown promising results in treating achondroplasia, the most common form of dwarfism, with significant improvements in annualized growth velocity (AGV) and secondary measures such as height Z-score and changes from baseline AGV.
BioMarin's VOXZOGO is currently the only approved drug for treating achondroplasia in the U.S. and Europe, but its daily dosing regimen may be less convenient compared to TransCon CNP's once-weekly dosing.

Competition and Market Impact:
The release of positive data from Ascendis Pharma's ApproaCH trial led to a 17% surge in Ascendis' stock price and a nearly 18% decline in BioMarin's shares, indicating a potential shift in market dominance.

Regulatory Plans:
Ascendis plans to file for regulatory approval for TransCon CNP with the FDA in the first quarter of 2025 and with the EMA by the third quarter of 2025.

Market Dynamics:
The presence of multiple drugs in development for achondroplasia, including those from BridgeBio Pharma and Pfizer, may influence reimbursement decisions in Europe, potentially affecting the market share of both VOXZOGO and TransCon CNP.