2023 ACMG Annual Clinical Genetics Meeting Digital Edition: Poster Gallery-Persistence of Growth Promoting Effects in Children with Achondroplasia Over Seven Years: Update from Phase II Extension Study with Vosoritide

Pdf Summary

This document summarizes the results of a study on the persistence of growth promoting effects in children with achondroplasia (a form of disproportionate short stature) over a period of seven years using the drug vosoritide. The study aimed to evaluate the safety and effectiveness of vosoritide in promoting growth in children with achondroplasia.<br /><br />The study included a total of 30 participants who received daily subcutaneous injections of vosoritide. The results showed that vosoritide was well-tolerated and there was no evidence of accelerated skeletal maturation or serious adverse events attributable to the drug over the seven-year treatment period.<br /><br />The study also found that vosoritide had a positive effect on growth. The annualized growth velocity (AGV) in children treated with vosoritide exceeded that seen in a reference population and approached that seen in an average stature population. There were sustained increases in height Z-scores over time, indicating an improvement in height compared to the untreated achondroplasia population. Additionally, the upper to lower body segment ratios continued to improve over time, especially in children aged below 11 years (girls) or below 12 years (boys).<br /><br />Overall, the study concluded that vosoritide had persistent growth promoting effects in children with achondroplasia over a seven-year period. The drug was well-tolerated and showed no evidence of accelerated skeletal maturation or serious adverse events. The findings support the ongoing use of vosoritide as a targeted therapy for achondroplasia.

Asset Subtitle

Submitter Only - Julie E. Hoover-Fong, MD, PhD; Presenting Author - Julie E. Hoover-Fong, MD, PhD; Co-Author - Melita Irving, MD; Co-Author - Carlos A. Bacino, MD, FACMG; Co-Author - Joel Charrow, MD; Co-Author - Valérie Cormier-Daire, MD, PhD; Co-Author - Lynda E. Polgreen, MD; Co-Author - Paul R. Harmatz, MD; Co-Author - Alice Huntsman-Labed, PhD; Co-Author - Elena Fisheleva, MD; Co-Author - Ian Sabir; Co-Author - Jonathan Day, MB, BS, PhD; Co-Author - John A. Phillips, III; Co-Author - Ravi Savarirayan, MD, PhD;

Meta Tag

Bone/Joint Abnormalities

Musculoskeletal system

Therapy

Co-Author Melita Irving, MD

Co-Author Carlos A. Bacino, MD, FACMG

Co-Author Joel Charrow, MD

Co-Author Valérie Cormier-Daire, MD, PhD

Co-Author Lynda E. Polgreen, MD

Co-Author Paul R. Harmatz, MD

Co-Author Alice Huntsman-Labed, PhD

Co-Author Elena Fisheleva, MD

Co-Author Ian Sabir

Co-Author Jonathan Day, MB, BS, PhD

Co-Author John A. Phillips, III

Co-Author Ravi Savarirayan, MD, PhD

Presenting Author Julie E. Hoover-Fong, MD, PhD

Submitter Only Julie E. Hoover-Fong, MD, PhD

Keywords

achondroplasia

growth promoting effects

vosoritide

persistence

children

study

safety

effectiveness

skeletal maturation

adverse events