P144: Persistence of growth-promoting effects in children with achondroplasia up to 7 years: Update from phase 2 extension study with vosoritide*
Year: 2024
Type: article
Abstract: Vosoritide is approved for use in children with achondroplasia (ACH) on the basis of a pivotal phase 3 study which demonstrated significant improvements in annualized growth velocity (AGV) vs placebo. Long-term data are being collected in the ongoing BMN 111-205 study, an extension to a dose-ranging phase 2 study (111-202) which was conducted in children aged 5–14 years at screening. Here we report safety and efficacy findings after a mean of 80.95 (SD 17.73, maximum 106.7) months exposure, representing the longest clinical experience with vosoritide to date.
Source: Genetics in Medicine Open
Authors: Julie Hoover-Fong, Melita Irving, Carlos Bacino, Joel Charrow, Carlos Prada, Valerie Cormier-Daire, Lynda Polgreen, Paul Harmatz, Sajda Ghani, Elena Fisheleva, Andrea Low, Jonathan Day, John Phillips, Ravi Savarirayan
Institutions: Johns Hopkins University, Johns Hopkins Medicine, Guy's and St Thomas' NHS Foundation Trust, Evelina London Children's Healthcare, Baylor College of Medicine, Lurie Children's Hospital, Hôpital Necker-Enfants Malades, University of California, Los Angeles, The Lundquist Institute, Harbor–UCLA Medical Center, UCSF Benioff Children's Hospital, BioMarin (United Kingdom), BioMarin (United States), Vanderbilt University Medical Center, Royal Children's Hospital, The University of Melbourne, Murdoch Children's Research Institute
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,200
OpenAlex ID: W4392606069