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Hypochondroplasia

2
Pipeline Programs
4
Companies
7
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
1
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide
360%
Small Molecule
240%
+ 5 programs with unclassified modality

Competitive Landscape

4 companies ranked by most advanced pipeline stage

QED Therapeutics
QED TherapeuticsCA - Palo Alto
3 programs
1
1
infigratinib 0.128 mg/kg/dayPhase 2/3
InfigratinibPhase 2Small Molecule
Prospective Clinical Assessment Study in Children With HypochondroplasiaN/A
BridgeBio Pharma
BridgeBio PharmaCA - Palo Alto
3 programs
Prospective Clinical Assessment Study in Children With HypochondroplasiaN/A1 trial
InfigratinibPHASE_2Small Molecule1 trial
infigratinib 0.128 mg/kg/dayPHASE_2_31 trial
Active Trials
NCT06410976Recruiting150Est. Oct 2026
NCT07393373Enrolling By Invitation135Est. May 2036
NCT06873035Enrolling By Invitation24Est. Jan 2027
BioMarin Pharmaceutical
3 programs
VosoritidePHASE_2Peptide1 trial
VosoritidePHASE_3Peptide1 trial
VosoritidePHASE_3Peptide1 trial
Active Trials
NCT07126262Recruiting60Est. Jun 2028
NCT06455059Active Not Recruiting80Est. Aug 2026
NCT07073014Enrolling By Invitation140Est. Dec 2040
Merck & Co.
Merck & Co.RAHWAY, NJ
1 program
Recombinant human growth hormonePHASE_21 trial
Active Trials
NCT01111019Completed19Est. Jan 2017

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
BioMarin PharmaceuticalVosoritide
BioMarin PharmaceuticalVosoritide
BridgeBio Pharmainfigratinib 0.128 mg/kg/day
BridgeBio PharmaInfigratinib
BioMarin PharmaceuticalVosoritide
Merck & Co.Recombinant human growth hormone
BridgeBio PharmaProspective Clinical Assessment Study in Children With Hypochondroplasia

Clinical Trials (7)

Total enrollment: 608 patients across 7 trials

Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

Start: Jun 2025Est. completion: Dec 2040140 patients
Phase 3Enrolling By Invitation

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Start: Jun 2024Est. completion: Aug 202680 patients
Phase 3Active Not Recruiting
NCT06873035BridgeBio Pharmainfigratinib 0.128 mg/kg/day

An Interventional Study of Infigratinib in Children With Hypochondroplasia

Start: Apr 2025Est. completion: Jan 202724 patients
Phase 2/3Enrolling By Invitation

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Start: Apr 2026Est. completion: May 2036135 patients
Phase 2Enrolling By Invitation

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Start: Jul 2025Est. completion: Jun 202860 patients
Phase 2Recruiting
NCT01111019Merck & Co.Recombinant human growth hormone

Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children

Start: Mar 2006Est. completion: Jan 201719 patients
Phase 2Completed
NCT06410976BridgeBio PharmaProspective Clinical Assessment Study in Children With Hypochondroplasia

Prospective Clinical Assessment Study in Children With Hypochondroplasia

Start: Jun 2024Est. completion: Oct 2026150 patients
N/ARecruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 actively recruiting trials targeting 608 patients
4 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.