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Achondroplasia

11
Pipeline Programs
10
Companies
33
Clinical Trials
9 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
1
7
1
1
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
250%
Peptide
250%
+ 30 programs with unclassified modality

On Market (1)

Approved therapies currently available

Ascendis Pharma
YUVIWELApproved
navepegritide
Ascendis Pharma
subcutaneous2026

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Ascendis Pharma
Ascendis PharmaCA - Palo Alto
3 programs
1
1
1
TransCon CNPPhase 2/34 trials
YUVIWEL(navepegritide)Phase 2Peptide2 trials
AchondroplasiaPhase N/A1 trial
Active Trials
NCT06732895Recruiting24Est. Sep 2027
NCT06079398Recruiting72Est. Dec 2028
NCT05929807Enrolling By Invitation140Est. Mar 2039
+4 more trials
QED Therapeutics
QED TherapeuticsCA - Palo Alto
5 programs
3
1
Infigratinib 0.25 mg/kg/dayPhase 3
InfigratinibPhase 2Small Molecule
Infigratinib 0.016 mg/kgPhase 2
Infigratinib is provided as a single dose of minitablets for oral administrationPhase 2
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)N/A
Pfizer
PfizerNEW YORK, NY
2 programs
1
ReciferceptPhase 21 trial
ReciferceptN/A1 trial
Active Trials
NCT05659719Completed248Est. May 2023
NCT04638153Terminated60Est. Mar 2023
Abbisko Therapeutics
Abbisko TherapeuticsChina - Shanghai
2 programs
1
1
ABSK061Phase 23 trials
ABSK061Phase 1/2
Active Trials
NCT07297875Not Yet Recruiting110Est. Mar 2031
NCT07007546Recruiting42Est. Nov 2025
NCT05244551Recruiting85Est. Apr 2025
Tyra Biosciences
Tyra BiosciencesCA - Carlsbad
1 program
1
TYRA-300 0.125 mg/kgPhase 21 trial
Active Trials
NCT06842355Recruiting92Est. Jun 2030
BioMarin Pharmaceutical
14 programs
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With AchondroplasiaN/A1 trial
Achondroplasia Natural History Multicenter Clinical StudyN/A1 trial
Lifetime Impact Study for AchondroplasiaN/A1 trial
Lifetime Impact of Achondroplasia Study in Europe-LIAISEN/A1 trial
VIrtual STudy in Achondroplasia for the US (VISTA)N/A1 trial
+9 more programs
Active Trials
NCT01603095Completed363Est. Feb 2021
NCT02597881Recruiting1,500Est. Dec 2030
NCT03872531Completed173Est. Feb 2021
+11 more trials
BridgeBio Pharma
BridgeBio PharmaCA - Palo Alto
5 programs
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)N/A1 trial
InfigratinibPHASE_2Small Molecule1 trial
Infigratinib 0.016 mg/kgPHASE_21 trial
Infigratinib is provided as a single dose of minitablets for oral administrationPHASE_21 trial
Infigratinib 0.25 mg/kg/dayPHASE_31 trial
Active Trials
NCT04035811Completed271Est. Aug 2025
NCT05145010Enrolling By Invitation300Est. Feb 2032
NCT04265651Completed84Est. Oct 2024
+2 more trials
Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
Issues Surrounding Prenatal Genetic Testing for AchondroplasiaN/A1 trial
Active Trials
NCT00001536Completed2,000Est. Jul 2000
Genome & Company
Genome & CompanyKorea - Suwon
1 program
Issues Surrounding Prenatal Genetic Testing for AchondroplasiaN/A

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
BridgeBio PharmaInfigratinib 0.25 mg/kg/day
BioMarin PharmaceuticalBMN 111
BioMarin PharmaceuticalBMN 111
BioMarin PharmaceuticalBMN 333
Ascendis PharmaTransCon CNP
Ascendis PharmaTransCon CNP
BridgeBio PharmaInfigratinib is provided as a single dose of minitablets for oral administration
Tyra BiosciencesTYRA-300 0.125 mg/kg
Ascendis Pharmanavepegritide
Ascendis Pharmanavepegritide
Ascendis PharmaTransCon CNP
BridgeBio PharmaInfigratinib
PfizerRecifercept
BioMarin Pharmaceuticalvosoritide
Ascendis PharmaTransCon CNP

Showing 15 of 33 trials with date data

Clinical Trials (33)

Total enrollment: 7,216 patients across 33 trials

NCT06164951BridgeBio PharmaInfigratinib 0.25 mg/kg/day

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

Start: Nov 2023Est. completion: Dec 2025114 patients
Phase 3Completed
NCT03424018BioMarin PharmaceuticalBMN 111

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Start: Dec 2017Est. completion: Jun 2031119 patients
Phase 3Active Not Recruiting
NCT03197766BioMarin PharmaceuticalBMN 111

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Start: Dec 2016Est. completion: Oct 2019121 patients
Phase 3Completed
NCT07441876BioMarin PharmaceuticalBMN 333

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Start: Apr 2026Est. completion: Sep 2029160 patients
Phase 2/3Recruiting
NCT05929807Ascendis PharmaTransCon CNP

A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

Start: Jun 2023Est. completion: Mar 2039140 patients
Phase 2/3Enrolling By Invitation
NCT05598320Ascendis PharmaTransCon CNP

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia

Start: Mar 2023Est. completion: Aug 202584 patients
Phase 2/3Completed
NCT07169279BridgeBio PharmaInfigratinib is provided as a single dose of minitablets for oral administration

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

Start: Nov 2025Est. completion: Mar 203277 patients
Phase 2Recruiting
NCT06842355Tyra BiosciencesTYRA-300 0.125 mg/kg

A Study of TYRA-300 in Children With Achondroplasia: BEACH301

Start: Mar 2025Est. completion: Jun 203092 patients
Phase 2Recruiting
NCT06732895Ascendis Pharmanavepegritide

A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.

Start: Dec 2024Est. completion: Sep 202724 patients
Phase 2Recruiting
NCT06079398Ascendis Pharmanavepegritide

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

Start: Jan 2024Est. completion: Dec 202872 patients
Phase 2Recruiting
NCT05246033Ascendis PharmaTransCon CNP

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia

Start: Jan 2022Est. completion: Mar 202424 patients
Phase 2Unknown
NCT05145010BridgeBio PharmaInfigratinib

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

Start: Dec 2021Est. completion: Feb 2032300 patients
Phase 2Enrolling By Invitation
NCT04638153PfizerRecifercept

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

Start: Dec 2020Est. completion: Mar 202360 patients
Phase 2Terminated
NCT04554940BioMarin Pharmaceuticalvosoritide

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia

Start: Oct 2020Est. completion: Dec 202720 patients
Phase 2Active Not Recruiting
NCT04085523Ascendis PharmaTransCon CNP

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

Start: Jun 2020Est. completion: Oct 202457 patients
Phase 2Completed
NCT04265651BridgeBio PharmaInfigratinib 0.016 mg/kg

Study of Infigratinib in Children With Achondroplasia

Start: Mar 2020Est. completion: Oct 202484 patients
Phase 2Completed
NCT03583697BioMarin PharmaceuticalBMN 111

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

Start: Jun 2018Est. completion: Jan 202275 patients
Phase 2Completed
NCT02724228BioMarin PharmaceuticalBMN 111

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

Start: Jan 2016Est. completion: Feb 202830 patients
Phase 2Active Not Recruiting
NCT02055157BioMarin PharmaceuticalBMN 111

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

Start: Jan 2014Est. completion: Oct 201735 patients
Phase 2Completed
NCT07297875Abbisko TherapeuticsABSK061

A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia

Start: Dec 2025Est. completion: Mar 2031110 patients
Phase 1/2Not Yet Recruiting
NCT07007546Abbisko TherapeuticsABSK061

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ABSK061 Mini-tablets in Healthy Adult Participants

Start: May 2025Est. completion: Nov 202542 patients
Phase 1Recruiting
NCT05813314BioMarin PharmaceuticalBMN 111 administration via Injector Pen

Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

Start: Mar 2023Est. completion: Jun 202336 patients
Phase 1Terminated
NCT05244551Abbisko TherapeuticsABSK061

A Study to Assess Safety, Tolerability, and Pharmacokinetics of ABSK061 in Patients With Advanced Solid Tumors

Start: Jun 2022Est. completion: Apr 202585 patients
Phase 1Recruiting
NCT01590446BioMarin PharmaceuticalBMN 111

A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

Start: Feb 2012Est. completion: Jun 201274 patients
Phase 1Completed
NCT06168201BioMarin PharmaceuticalVIrtual STudy in Achondroplasia for the US (VISTA)

VIrtual STudy in Achondroplasia for the US (VISTA)

Start: Feb 2023Est. completion: Feb 2028170 patients
N/ARecruiting
NCT05659719PfizerRecifercept

A Study to Learn About Recifercept in Patients With Achondroplasia

Start: Oct 2022Est. completion: May 2023248 patients
N/ACompleted
NCT04035811BridgeBio PharmaProspective Clinical Assessment Study in Children With Achondroplasia (ACH)

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Start: Aug 2019Est. completion: Aug 2025271 patients
N/ACompleted
NCT03875534Ascendis PharmaAchondroplasia

A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia

Start: Jun 2019Est. completion: Jan 2024260 patients
N/ACompleted
NCT03872531BioMarin PharmaceuticalLifetime Impact Study for Achondroplasia

Lifetime Impact Study for Achondroplasia

Start: Mar 2019Est. completion: Feb 2021173 patients
N/ACompleted
NCT03449368BioMarin PharmaceuticalLifetime Impact of Achondroplasia Study in Europe-LIAISE

Lifetime Impact of Achondroplasia Study in Europe-LIAISE

Start: Dec 2017Est. completion: Apr 2020196 patients
N/ACompleted
NCT02597881BioMarin PharmaceuticalAchondroplasia Natural History Multicenter Clinical Study

Achondroplasia Natural History Multicenter Clinical Study

Start: Apr 2016Est. completion: Dec 20301,500 patients
N/ARecruiting
NCT01603095BioMarin PharmaceuticalA Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

Start: Apr 2012Est. completion: Feb 2021363 patients
N/ACompleted
NCT00001536Human BioSciencesIssues Surrounding Prenatal Genetic Testing for Achondroplasia

Issues Surrounding Prenatal Genetic Testing for Achondroplasia

Start: Aug 1996Est. completion: Jul 20002,000 patients
N/ACompleted

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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

1 late-stage (Phase 3) programs, potential near-term approvals
9 actively recruiting trials targeting 7,216 patients
10 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.