Orphan Drug Designation Granted for TransCon CNP
The FDA grants orphan status to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the
We predict that EMA, the European Medicines Agency, will also grant orphan drug designation to TransCon CNP soon.
“We are pleased to receive Orphan Drug Designation for TransCon CNP in achondroplasia. Our TransCon CNP candidate is designed to address not only height but the debilitating comorbidities of the condition,” said
The phase 1 preliminary results were also mentioned (read more here), showing that TransCon CNP delivered continuous exposure of CNP at target levels over seven days, supporting once-weekly dosing with a well-tolerated safety profile.
Based on these preliminary results, Ascendis expects to initiate a phase 2 trial in children with achondroplasia in the third quarter of this year.
As an important step on the research and development of TransCon CNP, Ascendis is starting the ACHieve Study, an observational study that aims to provide important observational insights into the experience of children living with achondroplasia.
More news on the ACHive study, the EMA Orphan drug designation and Phase 2 clinical trial for TransCon CNP will be published in Beyond Achondroplasia as soon the company releases this information. Subscribe to our monthly newsletter here to receive these updates in your e-mail.