Ascendis Pharma has received the European Commission (EC) Orphan Designation for its TransCon C-Type Natriuretic Peptide (CNP) for the treatment of achondroplasia, the common form of dwarfism.

TransCon CNP is an investigational long-acting drug, designed to facilitate uninterrupted exposure of CNP at safe, therapeutic levels through a single, weekly subcutaneous dose.

The biopharmaceutical firm said that its TransCon CNP has previously received the US FDA orphan designation for the treatment of achondroplasia in February 2019.

Ascendis Pharma development operations senior vice president Dana Pizzuti said: “We now have orphan designations in Europe and the US for TransCon CNP in achondroplasia, which we believe signifies the importance of developing a safe and effective therapy that can address the many medical complications these children face.

“With our global phase 2 ACcomplisH Trial of TransCon CNP underway in achondroplasia, we are on track with the development of TransCon CNP and our receipt of the orphan designation in Europe is a key step towards developing a new treatment option for children with achondroplasia.”

TransCon comprises an unmodified parent drug, an inert carrier and a linker

Ascendis has designed its advanced TransCon (transient conjugation) platform technology to create new therapies that optimising therapeutic effect, including efficacy, safety and dosing frequency.

TransCon molecules constitute three parts, including an unmodified parent drug, an inert carrier to protect the parent drug, and a linker to temporarily bind them both.

When injected into the body, the active, unmodified parent drug will be released in a predictable manner, triggered by the physiologic conditions, as the parent drug is unmodified, and its original mode of action is maintained.

The company said that its TransCon technology has been designed for broad applications in various protein, peptide or small molecule therapeutic areas, for systemic or local use.

Achondroplasia is the most common form of dwarfism, where people living with the disease experience various skeletal complications and comorbidities.

Abnormal skeletal development may lead to sleep apnea, chronic back and leg pain from lower spine impingement and sudden infant death from cervical compression, and abnormal eustachian tubes may lead to chronic ear infections, resulting in hearing loss and speech delay.