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Evidence dashboardStudies since 2012Updated Jul 31, 2026

VosoritideClinical Trials.

A source-linked view of every Vosoritide study in the current snapshot—status, phase, enrollment, results, publications, locations and changes over time.

Total trials
18
Recruiting
5
Completed
4
Terminated
1
Results posted
3
Average enrollment
69
Countries
13
Highest phase
Phase 3

Largest indexed study

160 participants · NCT07441876 →

Developer / research origin

BioMarin

Evidence summary

Activity is not efficacy.

Vosoritide appears in 18 indexed studies: 5 recruiting, 4 completed and 3 with registry results posted. The highest registered development stage in this snapshot is Phase 3. These counts measure registry activity; they do not establish that Vosoritide is safe or effective.

Review the design, endpoints, population, protocol changes, statistical analysis and publications before drawing conclusions.

Development history

Trial timeline.

Evidence timeline

2012—2026

201220142016201720182024202420242024202520252026

Velocity

Vosoritide studies by start year

Study start year · indexed snapshot

4321020122012: 1 trials2013: 0 trials2014: 1 trials20152015: 0 trials2016: 2 trials2017: 1 trials20182018: 1 trials2019: 1 trials2020: 2 trials20212021: 0 trials2022: 0 trials2023: 2 trials20242024: 4 trials2025: 2 trials20262026: 1 trials

Development

Trials by phase

Early Phase 10
Phase 13
Phase 212
Phase 35
Phase 40

Current studies

Recruiting or active

Completed studies

Finished records

Stopped studies

Withdrawn or terminated

Showing 6 of 13 studies

Full study index

Search Vosoritide trials.

Add status, phase, condition, country, result and publication filters inside this molecule’s indexed study set.

18 matching studies

End of 18 results

Latest publications

Follow the paper trail.

PMID 38813446 ↗Dauber A, Zhang A, Kanakatti Shankar R, Boucher K, McCarthy T, Shafaei N, Seaforth R, Castro MG, Dham N, Merchant N. Vosoritide treatment for children with hypochondroplasia: a phase 2 trial. EClinicalMedicine. 2024 Apr 11;71:102591. doi: 10.1016/j.eclinm.2024.102591. eCollection 2024 May.Linked from NCT04219007PMID 38649657 ↗Qi Y, Chan ML, Mould DR, Larimore K, Fisheleva E, Cherukuri A, Day J, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Bober MB, Henshaw J. Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clin Pharmacokinet. 2024 May;63(5):707-719. doi: 10.1007/s40262-024-01371-6. Epub 2024 Apr 23.Linked from NCT02724228PMID 31269546 ↗Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18.Linked from NCT02724228PMID 39740666 ↗Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Palm K, Prada CE, Kubota T, Arundel P, Kotani Y, Leiva-Gea A, Bober MB, Hecht JT, Legare JM, Lawrinson S, Low A, Sabir I, Huntsman-Labed A, Day JRS. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. Med. 2025 May 9;6(5):100566. doi: 10.1016/j.medj.2024.11.019. Epub 2024 Dec 30.Linked from NCT03424018PMID 39305160 ↗Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Mohnike K, Prada CE, Kubota T, Arundel P, Leiva-Gea A, Rowell R, Low A, Sabir I, Huntsman-Labed A, Day J. Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life. Genet Med. 2024 Dec;26(12):101274. doi: 10.1016/j.gim.2024.101274. Epub 2024 Sep 18.Linked from NCT03424018PMID 33761804 ↗Savarirayan R, Irving M, Maixner W, Thompson D, Offiah AC, Connolly DJ, Raghavan A, Powell J, Kronhardt M, Jeha G, Ghani S, Fisheleva E, Day JR. Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery. Sci Prog. 2021 Jan-Mar;104(1):368504211003782. doi: 10.1177/00368504211003782.Linked from NCT04554940PMID 41783511 ↗Irving M, Savarirayan R, Hoover-Fong JE, Dee A, Mukherjee S, Rivat C, Sabir I, White KK. Effect of vosoritide on spine morphology in children with achondroplasia: 1-year results from a randomized phase 2 study. J Endocr Soc. 2026 Feb 20;10(3):bvag008. doi: 10.1210/jendso/bvag008. eCollection 2026 Mar.Linked from NCT03583697PMID 37984383 ↗Savarirayan R, Wilcox WR, Harmatz P, Phillips J 3rd, Polgreen LE, Tofts L, Ozono K, Arundel P, Irving M, Bacino CA, Basel D, Bober MB, Charrow J, Mochizuki H, Kotani Y, Saal HM, Army C, Jeha G, Qi Y, Han L, Fisheleva E, Huntsman-Labed A, Day J. Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Child Adolesc Health. 2024 Jan;8(1):40-50. doi: 10.1016/S2352-4642(23)00265-1. Epub 2023 Nov 18.Linked from NCT03583697

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