Hypochondroplasia

7
Pipeline Programs
3
Companies
7
Clinical Trials
2 recruiting
1
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

Peptide
375%
Small Molecule
125%
+ 3 programs with unclassified modality

On Market (1)

Approved therapies currently available

BioMarin Pharmaceutical
VOXZOGOApproved
vosoritide
BioMarin Pharmaceutical
subcutaneous2021

Competitive Landscape

3 companies ranked by most advanced pipeline stage

BioMarin Pharmaceutical
3 programs
1
2
1
VosoritidePhase 3Peptide1 trial
VosoritidePhase 3Peptide1 trial
VosoritidePhase 2Peptide1 trial
Active Trials
NCT07126262Recruiting60Est. Jun 2028
NCT06455059Active Not Recruiting80Est. Aug 2026
NCT07073014Enrolling By Invitation140Est. Dec 2040
BridgeBio Pharma
BridgeBio PharmaCA - Palo Alto
3 programs
1
1
infigratinib 0.128 mg/kg/dayPhase 2/31 trial
InfigratinibPhase 2Small Molecule1 trial
Prospective Clinical Assessment Study in Children With HypochondroplasiaN/A1 trial
Active Trials
NCT06410976Recruiting150Est. Oct 2026
NCT07393373Enrolling By Invitation135Est. May 2036
NCT06873035Enrolling By Invitation24Est. Jan 2027
M&
Merck & Co.RAHWAY, NJ
1 program
1
Recombinant human growth hormonePhase 21 trial
Active Trials
NCT01111019CompletedEst. 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: 589 patients across 7 trials

NCT07073014BioMarin PharmaceuticalVosoritide

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

Start: Jun 2025Est. completion: Dec 2040140 patients
Phase 3Enrolling By Invitation
NCT06455059BioMarin PharmaceuticalVosoritide

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
NCT07393373BridgeBio PharmaInfigratinib

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

Start: Apr 2026Est. completion: May 2036135 patients
Phase 2Enrolling By Invitation
NCT07126262BioMarin PharmaceuticalVosoritide

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 2017
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

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
2 actively recruiting trials targeting 589 patients
3 companies competing in this space