2023 ACMG Annual Clinical Genetics Meeting Digital Edition: Poster Gallery-Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 3.5 years: update from Phase 3 extension study

Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 3.5 years: update from Phase 3 extension study

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This study presented the results of an ongoing Phase 3 extension study evaluating the effects of vosoritide in children with achondroplasia, a form of disproportionate short stature caused by a specific genetic variant. The study included a total of 119 participants who received daily subcutaneous injections of vosoritide for up to 3.5 years.<br /><br />The study found that treatment with vosoritide resulted in persistent and stable growth promoting effects in children with achondroplasia. The average age of the participants at the start of treatment was 9.18 years, and the majority of participants were between the ages of 5 and 11 years. There were slightly more male participants than female participants.<br /><br />The results showed that there was a consistent increase in growth velocity with vosoritide treatment compared to untreated children with achondroplasia. The difference in growth velocity between treated and untreated children was higher in males compared to females. There was also a significant improvement in height z-score, which measures how a child's height compares to a reference population of children with achondroplasia, over time with vosoritide treatment.<br /><br />The study also demonstrated that vosoritide was generally well-tolerated with a favorable safety profile. The most common adverse event was injection site reactions, which were mostly mild and self-limiting. There were no serious adverse events related to the treatment, and no subjects discontinued treatment due to an adverse event.<br /><br />Overall, the study showed that vosoritide treatment in children with achondroplasia resulted in sustained and significant increases in growth velocity and improvement in height compared to untreated children. The treatment was well-tolerated and had a favorable safety profile. These findings highlight the potential of vosoritide as a targeted therapy for achondroplasia.

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Co-Author - Julie E. Hoover-Fong, MD, PhD; Presenting Author - Julie E. Hoover-Fong, MD, PhD; Co-Author - Ravi Savarirayan, MD, PhD; Co-Author - Louise Tofts, MBBS; Co-Author - Melita Irving, MD; Co-Author - William R. Wilcox, MD, PhD, FACMG; Co-Author - Carlos A. Bacino, MD, FACMG; Co-Author - Rosendo Ullot Font, MD; Co-Author - Paul R. Harmatz, MD; Co-Author - Frank Rutsch, MD; Co-Author - Michael B. Bober, MD; Co-Author - Lynda E. Polgreen, MD; Co-Author - Ignacio Ginebreda, MD; Co-Author - Klaus Mohnike, MD; Co-Author - Joel Charrow, MD; Co-Author - Daniel Hoernschemeyer, MD; Co-Author - Keiichi Ozono, MD; Co-Author - Yasemin Alanay, MD, PhD; Co-Author - Paul Arundel, MD; Co-Author - Shoji Kagami, MD; Co-Author - Natsuo Yasui, MD; Co-Author - Klane K. White, MD; Co-Author - Howard M. Saal, MD, FACMG, FAAP; Co-Author - Antonio Leiva-Gea, MD; Co-Author - Felipe Luna-González, MD; Co-Author - Hiroshi Mochizuki, MD; Co-Author - Donald Basel, MBBCh; Co-Author - Dania M. Porco, MS, MBA; Co-Author - Kala Jayaram, MD; Co-Author - Elena Fisheleva, MD; Co-Author - Sue Lawrinson, PhD; Co-Author - Jonathan Day, MB, BS, PhD;

Meta Tag

Bone/Joint Abnormalities

Musculoskeletal system

Therapy

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

Co-Author Ravi Savarirayan, MD, PhD

Co-Author Louise Tofts, MBBS

Co-Author Melita Irving, MD

Co-Author William R. Wilcox, MD, PhD, FACMG

Co-Author Carlos A. Bacino, MD, FACMG

Co-Author Rosendo Ullot Font, MD

Co-Author Paul R. Harmatz, MD

Co-Author Frank Rutsch, MD

Co-Author Michael B. Bober, MD

Co-Author Lynda E. Polgreen, MD

Co-Author Ignacio Ginebreda, MD

Co-Author Klaus Mohnike, MD

Co-Author Joel Charrow, MD

Co-Author Daniel Hoernschemeyer, MD

Co-Author Keiichi Ozono, MD

Co-Author Yasemin Alanay, MD, PhD

Co-Author Paul Arundel, MD

Co-Author Shoji Kagami, MD

Co-Author Natsuo Yasui, MD

Co-Author Klane K. White, MD

Co-Author Howard M. Saal, MD, FACMG, FAAP

Co-Author Antonio Leiva-Gea, MD

Co-Author Felipe Luna-González, MD

Co-Author Hiroshi Mochizuki, MD

Co-Author Donald Basel, MBBCh

Co-Author Dania M. Porco, MS, MBA

Co-Author Kala Jayaram, MD

Co-Author Elena Fisheleva, MD

Co-Author Sue Lawrinson, PhD

Co-Author Jonathan Day, MB, BS, PhD

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

Keywords

Phase 3 extension study

vosoritide

achondroplasia

disproportionate short stature

genetic variant

growth promoting effects

subcutaneous injections

growth velocity

height z-score

safety profile