Biotech Industry Liaison Committee Review

1About achondroplasia. National Institute of Health. (2016, July). Retrieved September 2021, from National Institute of Health - Achondroplasia; July 2016.
2Wright, MR, et al. Clinical Management of Achondroplasia. Arch Dis Child. 2012 Feb;97(2):129-34.
3Achondroplasia.Little People of America. Retrieved September 2021, from
4Holler, W, et al. New developments in the management of achondroplasia. 2020 Apr;170(5-6):104-111
5What is Achondroplasia. Biomarin. Retrieved September 2021 from
6The Drug Development Process. US Food & Drug Administration. (2018, January). Retrieved September 2021, from
7National Library of Medicine (U.S.). Clinical Trials - Achondroplasia. National Institute of Health. Retrieved September 2021.
8Biomarin Press Releases. Biomarin. Retrieved September 2021 from
9Savarirayan, R, et al. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomized, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet. 2020 Sep 5;396(10252):684- 692.
10Savarirayan, R, et al. Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genet Med (2021).
11Breinholt, VM, et al. TransCon CNP, a Sustained-Release C-Type Natriuretic Peptide Prodrug, a Potentially Safe and Efficacious New Therapeutic Modality for the Treatment of Comorbidities Associated with Fibroblast Growth Factor Receptor 3-Related Skeletal Dysplasias. J Pharmacol Exp Ther. 2019 Sep;370(3):459-471. DOI:
12National Library of Medicine (U.S.). (2020-2026). A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia. Identifier NCT04085523.
13Savarirayan, R. et al. Infigratinib in Children With Achondroplasia (ACH): Design of PROPEL2 - A Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study, Journal of the Endocrine Society, Volume 5, Issue Supplement_1, AprilMay 2021, Pages A667–A668,
14National Library of Medicine (U.S.). (2020-2023). Study of Infigratinib in Children With Achondroplasia. Identifier NCT04265651.
15Gonçalves, D, et al. In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia. PLoS One. 2020 Dec 28;15(12):e0244368.
16National Library of Medicine (U.S.). (2020-2023). A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia Identifier NCT04638153.