BioMarin股价下跌17%，因为Ascendis数据被视为对Voxzogo的威胁

2024-09-17 02:59

BioMarin Pharmaceutical (NASDAQ:BMRN) is down ~17% Monday following positive data from an Ascendis Pharma (ASND) achondroplasia candidate that could challenge the former's Voxzogo.
Data from the pivotal ApproaCH trial of TransCon CNP (navepegritide) showed the treatment led to annualized growth velocity (AGV) that was better than placebo. At week 52, children given TransCon CNP demonstrated a mean AGV of 5.89 cm/year compared to 4.41 cm/year in the placebo group.
In addition, the once-weekly injection also led to improvements in height Z-score and change from baseline AGV.
"TransCon CNP-treated children with achondroplasia exceeded the growth rate of the general population, suggesting that once-weekly TransCon CNP provides catch-up growth with safety and tolerability results comparable to placebo with a low incidence of injection-site reactions,” Ascendis Jan Mikkelsen CEO said in a statement.
If approved, TransCon CNP would have a key dosing advantage over BioMarin's Voxzogo (vosoritide) given that the latter is given daily.
Ascendis is up ~19% on the data.

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