Ascendis Pharma (NASDAQ:ASND) has announced it has filed an investigational new drug application with the US Food and Drug Administration (FDA) to start its AComplisH Trial of TransCon CNP in children with achondroplasia.
As quoted in the press release:
“With the phase 2 ACcomplisH Trial, we aim to demonstrate that once-weekly TransCon CNP, which provides continuous exposure to CNP, is a potential therapeutic option for children with achondroplasia,” said Jonathan Leff, M.D., Chief Medical Officer of Ascendis Pharma. “By delivering continuous levels of CNP, TransCon CNP is expected to help restore balance to the fibroblast growth factor receptor 3 pathway, thereby addressing not only height but the many skeletal complications and comorbidities that can have life-altering implications for children with achondroplasia. Our hope is to offer a potential new therapeutic option that addresses these challenges to improve the overall health and well-being of children with achondroplasia.”
TransCon CNP is a long-acting prodrug of C-type natriuretic peptide (CNP) in development for children with achondroplasia. Phase 1 results demonstrated that TransCon CNP provided continuous exposure to CNP with a pharmacokinetic profile designed to maximize efficacy with once-weekly dosing. TransCon CNP was generally well tolerated at doses up to 150 µg/kg, with no serious adverse events or anti-CNP antibodies reported. TransCon CNP received Orphan Drug Designation (ODD) from the U.S. FDA in February 2019.
Achondroplasia is the most common form of dwarfism for which there is no FDA-approved therapy. Individuals living with achondroplasia may experience severe skeletal complications and comorbidities. For example, abnormal development of the vertebra can lead to sleep apnea, chronic back and leg pain from lower spine impingement and sudden infant death from cervical compression. Chronic ear infections due to abnormal eustachian tubes can lead to hearing loss and speech delay.