Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by their core values of patients, science and passion, they use their TransCon® technology to fulfill our mission of developing new and potentially best-in-class therapies that address unmet medical needs.
Ascendis is conducting the global ACHieve Study as one way to inform their development of TransCon CNP and learn about the experiences of children living with this rare condition. CNP has been shown to counteract the growth-inhibiting effects of the FGFR3 mutation and stimulate growth. Increasing CNP levels to modulate excessive FGFR3 signaling and rebalance the pathways that regulate growth may reduce the complications and comorbidities of ACH.
TransCon CNP is an investigational long-acting prodrug of CNP in development for the treatment of ACH in children. Designed to provide continuous CNP exposure, the goal of TransCon CNP is to optimize efficacy with a safe and convenient once-weekly dose. TransCon CNP has orphan designation for the treatment of ACH in both the U.S and Europe.
For more information on Ascendis’ commitment to understanding achondroplasia, visit their website: AscendisPharma.com