Orphan Drug Designation Granted for TransCon CNP

Ascendis Pharma A/S announced on the 28th February 2019, on the rare disease day that the U.S. Food and Drug Administration, FDA, has granted the Orphan Drug Designation (ODD) to TransCon CNP, a long-acting prodrug of C-type natriuretic peptide (CNP) in development for children with achondroplasia. Read the full press release here.

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 United States. The ODD program provides a drug developer with certain benefits and incentives but also requires that the company presents the safety and effectiveness results of TransCon CNP through adequate and well-controlled studies in order to support approval and commercialization. 

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 Jonathan Leff, M.D., Ascendis Pharma’s Chief Medical Officer.
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.