- BioMarin Pharmaceuticals (NASDAQ:BMRN) is Down ~17% On Monday, it became known that an achondroplasia candidate from Ascendis Pharma (ASND) reported positive data that could challenge predecessor Voxzogo.
- Data from the pivotal ApproaCH trial of TransCon CNP (navepegritide) showed that treatment produced better annualized growth velocity (AGH) than placebo. At week 52, children The TransCon CNP demonstrated a mean AGV of 5.89 cm/year compared with 4.41 cm/year in the placebo group.
- Furthermore, once-weekly injection also produced improvements in height Z-score and a change from baseline AGV.
- “Children with achondroplasia treated with TransCon CNP outperformed the general population's growth rate, suggesting that weekly administration of TransCon CNP allows for catch-up growth with comparable safety and tolerability outcomes to placebo and with a low incidence of injection site reactions,” Jan Mikkelsen, CEO of Ascendis, said in a statement.
- If approved, TransCon CNP would have a key dosing advantage over BioMarin's Voxzogo (vosoritide), given that the latter is administered daily.
- you approach him up to ~19% about the data.