References for Pharmaceutical Overview for Achondroplasia

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¹ Little People of America. Biotech Industry Liaison Committee. Accessed April 28, 2026. https://www.lpaonline.org/biotech-industry-liaison-committee
² Little People of America. Core Values. Accessed April 28, 2026. Core Values
³ BioMarin Pharmaceutical Inc. VOXZOGO (vosoritide) prescribing information. 2021. Accessed April 28, 2026. This label may not be the latest approved by FDA. For current labeling information, please visit https://www.fda.gov/drugsatfda
⁴ Ascendis Pharma Endocrinology, Inc. YUVIWEL (navepegritide) prescribing information. February 2026. Accessed April 28, 2026. This label may not be the latest approved by FDA. For current labeling information, please visit https://www.fda.gov/drugsatfda
⁵ U.S. Food and Drug Administration. Integrated review for NDA 214938: Vosoritide (VOXZOGO). 2021:51-53. Accessed April 28,  2026. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf
⁶ Savarirayan R, et al. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med. 2025;6(5):100566. doi:10.1016/j.medj.2024.11.019
⁷ BioMarin Pharmaceutical Inc. Achondroplasia: congress posters and presentations. Accessed April 28, 2026. https://medical.biomarin.com/en-us/achondroplasia/congress-posters-and-presentations/
⁸ Savarirayan R, McDonnell C, Bacino CA, et al. Once-weekly navepegritide in children with achondroplasia: the APPROACH randomized clinical trial. JAMA Pediatr. 2026;180(1):18-25. doi:10.1001/jamapediatrics.2025.4771
⁹ BridgeBio Pharma Inc. BridgeBio reports positive Phase 3 topline results for oral infigratinib with the first statistically significant improvements in body proportionality in achondroplasia. Published February 12, 2026. Accessed April 28, 2026. https://investor.bridgebio.com/news/news-details/2026/BridgeBio-Reports-Positive-Phase-3-Topline-Results-for-Oral-Infigratinib-with-the-First-Statistically-Significant-Improvements-in-Body-Proportionality-in-Achondroplasia/default.aspx
¹⁰ Savarirayan, R, De Bergua JM, Arundel P, et al. Oral infigratinib therapy in children with achondroplasia. N Engl J Med.2025;392(9):865-874. doi:10.1056/NEJMoa2411790
¹¹ Tyra Biosciences Inc. Tyra Biosciences announces first child dosed in BEACH301, its Phase 2 study for dabogratinib (TYRA-300) in pediatric achondroplasia. Published August 21, 2025. Accessed April 28, 2026. https://ir.tyra.bio/news-releases/news-release-details/tyra-biosciences-announces-first-child-dosed-beach301-its-phase
¹² Pauli RM. Achondroplasia: a comprehensive clinical review. Orphanet J Rare Dis. 2019;14:1. doi:10.1186/s13023-018-0972-6
¹³ Hoover-Fong J, Cheung MS, Fano V, et al. Lifetime impact of achondroplasia: current evidence and perspectives on the natural history. Bone. 2021;146:115872. doi:10.1016/j.bone.2021.115872
¹⁴ Hoover-Fong J, Scott CI, Jones MC; Committee on Genetics. Health supervision for people with achondroplasia. Pediatrics. 2020;145(6):e20201010. doi:10.1542/peds.2020-1010
¹⁵ Savarirayan R, Tofts L, Irving M, et al. International consensus statement on the diagnosis, multidisciplinary management, and lifelong care of individuals with achondroplasia. Nat Rev Endocrinol. 2022;18(3):173-189. doi:10.1038/s41574-021-00595-x
¹⁶ BridgeBio Pharma Inc. MyAchonJourney: resources and support for families living with achondroplasia. Accessed April 28, 2026.https://myachonjourney.com/
¹⁷ U.S. Food and Drug Administration. Accelerated Approval Program. Accessed April 28, 2026. https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approval-program
¹⁸ Ascendis Pharma. New data from Week 52 of the ongoing COACH trial showed that TransCon hGH accelerated TransCon CNP’s benefits beyond linear growth in children with achondroplasia. April 8, 2026. https://investors.ascendispharma.com/news-releases/news-release-details/new-data-week-52-ongoing-coach-trial-showed-transconr-hgh
¹⁹ Hudkins RL, Allen E, Balcer A, et al. Discovery of TYRA-300: First oral selective FGFR3 inhibitor for the treatment of urothelial cancers and achondroplasia. J Med Chem. 2024;67(18):16737–16756
²⁰ BioMarin Medical. Achondroplasia Publications. BioMarin Pharmaceutical Inc. Accessed July 19, 2026. https://medical.biomarin.com/en-us/achondroplasia/publications/
²¹ Savarirayan R, Hoover-Fong J, Irving M, et al. Phase 3 trial of oral infigratinib in children with achondroplasia. N Engl J Med. 2026. doi:10.1056/NEJMoa2604565
²² Savarirayan R, Tofts L, Irving M, et al. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet. 2020;396(10252):684-692. doi:10.1016/S0140-6736(20)31541-5

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