Ascendis Pharma Growth Disorders A/S

Address: Tuborg Boulevard 12

Sponsor Overview

assignment

Specializations

Achondroplasia in Children and Adolescents

Ascendis Pharma Growth Disorders A/S focuses its clinical research on achondroplasia across pediatric age groups, with studies centered on growth, linear growth, and the clinical needs of affected children and adolescents.

Growth outcomes
Pediatric skeletal dysplasia
Children with achondroplasia

The sponsor’s research activity includes both younger and older pediatric populations, reflecting a sustained interest in the treatment of growth disorders associated with this condition.

Growth-Targeted Therapeutics

The clinical portfolio includes investigational work on navepegritide and TransCon CNP, with a therapeutic emphasis on supporting growth in individuals with achondroplasia.

Linear growth
Growth promotion
C-type natriuretic peptide pathway

These studies place endocrine and skeletal growth mechanisms at the center of the sponsor’s research interests.

Combination Approaches in Growth Disorder Care

Research also extends to combined use of navepegritide with lonapegsomatropin, indicating interest in therapeutic strategies for improving height gain and growth response in pediatric achondroplasia.

Combination therapy
Somatropin-based treatment
Pediatric height development

This area reflects attention to treatment options that address the clinical burden of short stature in affected patients.

Safety, Tolerability, and Long-Term Use

Several trials examine the safety, tolerability, and sustained use of TransCon CNP and navepegritide in pediatric populations with achondroplasia.

Long-term treatment
Pediatric safety
Chronic growth management

The sponsor’s research interests include ongoing evaluation of treatment use over extended periods in children and adolescents.

Sponsored Trials

Trial Name	Country	Status
AttaCH: A Phase 2, Multicenter, Long-Term, Open Label Extension Trial Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children and Adolescents with Achondroplasia	Austria Denmark Finland France Germany Ireland Norway Portugal Sweden	Recruiting
Efficacy and Safety Evaluation of Navepegritide and Lonapegsomatropin in Pediatric Achondroplasia: A 156-Week, Phase 2, Open-Label, Single-Arm Study	Denmark Ireland	Not Recruiting
Evaluation of Efficacy and Safety of Subcutaneous Navepegritide in Adolescents with Achondroplasia: A Phase 2b Randomized, Double-Blind, Placebo-Controlled Trial	Denmark France Ireland	Recruiting
Phase 2 Multicenter Double-Blind Randomized Placebo-Controlled Trial of TransCon CNP in Infants with Achondroplasia Evaluating Safety, Tolerability, and Efficacy	Austria Denmark Finland France Germany Ireland Italy Norway Portugal Spain Sweden	Recruiting
Phase 2b Randomized, Double-Blind, Placebo-Controlled Trial of TransCon CNP in Pediatric Achondroplasia for Growth Efficacy and Safety Evaluation	Denmark Ireland Spain	Not Recruiting