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Achondroplasia

12
Pipeline Programs
11
Companies
34
Clinical Trials
7 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
8
1
1
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
250%
Peptide
250%
+ 31 programs with unclassified modality

On Market (1)

Approved therapies currently available

Ascendis Pharma
YUVIWELApproved
navepegritide
Ascendis Pharma
subcutaneous2026

Competitive Landscape

10 companies ranked by most advanced pipeline stage

Ascendis Pharma
Ascendis PharmaCA - Palo Alto
3 programs
1
1
1
TransCon CNPPhase 2/32 trials
YUVIWEL(navepegritide)Phase 2Peptide3 trials
AchondroplasiaPhase N/A1 trial
Active Trials
NCT06732895Recruiting24Est. Sep 2027
NCT06079398Recruiting72Est. Dec 2028
NCT05598320Completed84Est. Aug 2025
+3 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/A2 trials
Active Trials
NCT05659719Completed248Est. May 2023
NCT05116046Terminated35Est. Mar 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
NCT07007546Completed42Est. Apr 2026
NCT05244551Unknown85Est. Apr 2025
Tyra Biosciences
Tyra BiosciencesCA - Carlsbad
1 program
1
TYRA-300 0.125 mg/kgPhase 21 trial
Active Trials
NCT06842355Recruiting92Est. Jun 2030
VISEN Pharmaceuticals
VISEN PharmaceuticalsChina - Shanghai
1 program
1
TransCon CNPPhase 21 trial
Active Trials
NCT05246033Completed24Est. Apr 2024
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/A
Genome & Company
Genome & CompanyKorea - Suwon
1 program
Issues Surrounding Prenatal Genetic Testing for AchondroplasiaN/A1 trial
Active Trials
NCT00001536Completed2,000Est. Jul 2000

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 Pharmanavepegritide
BridgeBio PharmaInfigratinib is provided as a single dose of minitablets for oral administration
Tyra BiosciencesTYRA-300 0.125 mg/kg
Ascendis Pharmanavepegritide
Ascendis Pharmanavepegritide
VISEN PharmaceuticalsTransCon CNP
PfizerRecifercept
BridgeBio PharmaInfigratinib
PfizerRecifercept
BioMarin Pharmaceuticalvosoritide

Showing 15 of 34 trials with date data

Clinical Trials (34)

Total enrollment: 7,251 patients across 34 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 Pharmanavepegritide

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
NCT05246033VISEN PharmaceuticalsTransCon 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: Apr 202424 patients
Phase 2Completed
NCT05116046PfizerRecifercept

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

Start: Dec 2021Est. completion: Mar 202335 patients
Phase 2Terminated
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: Apr 202642 patients
Phase 1Completed
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 1Unknown
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
NCT00001536Genome & CompanyIssues Surrounding Prenatal Genetic Testing for Achondroplasia

Issues Surrounding Prenatal Genetic Testing for Achondroplasia

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

Related Jobs

Director, Marketing – Achondroplasia Community

BridgeBio PharmaBridgeBio Pharma
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Director, Marketing – Achondroplasia Community

QED TherapeuticsQED Therapeutics
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Aspa TherapeuticsAspa Therapeutics
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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
7 actively recruiting trials targeting 7,251 patients
11 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.