Biotech Industry Liaison Committee Review

References:
¹About achondroplasia. National Institute of Health. (2016, July). Retrieved September 2021, from https://www.genome.gov/Genetic-Disorders/Achondroplasia. National Institute of Health - Achondroplasia; July 2016.
²Wright, MR, et al. Clinical Management of Achondroplasia. Arch Dis Child. 2012 Feb;97(2):129-34. https://pubmed.ncbi.nlm.nih.gov/21460402/.
³Achondroplasia.Little People of America. Retrieved September 2021, from https://lpamrs.memberclicks.net/index.php?option=com_content&view=article&id=18.
⁴Holler, W, et al. New developments in the management of achondroplasia. 2020 Apr;170(5-6):104-111 https://pubmed.ncbi.nlm.nih.gov/32144686/.
⁵What is Achondroplasia. Biomarin. Retrieved September 2021 from  https://www.achondroplasia.com/what-is-achondroplasia.
⁶The Drug Development Process. US Food & Drug Administration. (2018, January). Retrieved September 2021, from https://www.fda.gov/patients/learnabout-drug-and-device-approvals/drug-development-process.
⁷National Library of Medicine (U.S.). Clinical Trials - Achondroplasia. National Institute of Health. Retrieved September 2021.
⁸Biomarin Press Releases. Biomarin. Retrieved September 2021 from https://investors.biomarin.com/2021-06-25-BioMarin-Receives-Positive-CHMPOpinion-in-Europe-for-Vosoritide-for-the-Treatment-of-Children-withAchondroplasia-from-Age-2-Until-Growth-Plates-Close.
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. https://doi.org/10.1016/S0140-6736(20)31541-5.
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). https://doi.org/10.1038/s41436-021-01287-7.
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: https://doi.org/10.1124/jpet.119.258251.
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. https://clinicaltrials.gov/ct2/show/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, https://doi.org/10.1210/jendso/bvab048.136.
¹⁴National Library of Medicine (U.S.). (2020-2023). Study of Infigratinib in Children With Achondroplasia. Identifier NCT04265651. https://clinicaltrials.gov/ct2/show/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. https://doi.org/10.1371/journal.pone.0244368.
¹⁶National Library of Medicine (U.S.). (2020-2023). A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia Identifier NCT04638153. https://clinicaltrials.gov/ct2/show/NCT04638153.