Achondroplasia

Pipeline Programs

Companies

Clinical Trials

4 recruiting

Approved Products

Pipeline by Development Stage

Preclinical

Phase 1

Phase 1/2

Phase 2

Phase 2/3

Phase 3

On Market

Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Peptide

375%

Small Molecule

125%

+ 24 programs with unclassified modality

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Pre

P1/2

P2/3

Mkt

BioMarin PharmaceuticalNOVATO, CA

14 programs

BMN 111Phase 31 trial

BMN 333Phase 2/31 trial

BMN 111Phase 21 trial

+9 more programs

Active Trials

NCT01603095Completed363Est. Feb 2021

NCT02597881Suspended1,500Est. Dec 2030

NCT03872531Completed173Est. Feb 2021

+11 more trials

BridgeBio PharmaCA - Palo Alto

5 programs

Infigratinib 0.25 mg/kg/dayPhase 31 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

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)N/A1 trial

Active Trials

NCT04035811Active Not Recruiting271Est. Jun 2026

NCT05145010Enrolling By Invitation300Est. Feb 2032

NCT04265651Completed84Est. Oct 2024

+2 more trials

Ascendis PharmaCA - Palo Alto

7 programs

TransCon CNPPhase 2/31 trial

Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injectionsPhase 21 trial

NavepegritidePhase 2Peptide1 trial

+2 more programs

Active Trials

NCT06433557Active Not Recruiting22Est. Nov 2027

NCT06079398Recruiting72Est. Dec 2028

NCT06732895Recruiting24Est. Jan 2027

+4 more trials

PfizerNEW YORK, NY

2 programs

ReciferceptPhase 21 trial

ReciferceptN/A1 trial

Active Trials

NCT05659719Completed248Est. May 2023

NCT04638153Terminated60Est. Mar 2023

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 333

Ascendis PharmaTransCon CNP

BridgeBio PharmaInfigratinib is provided as a single dose of minitablets for oral administration

Ascendis PharmaNavepegritide

Ascendis PharmaCombination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections

Ascendis PharmaNavepegritide

Ascendis PharmaTransCon CNP

BridgeBio PharmaInfigratinib

PfizerRecifercept

BioMarin Pharmaceuticalvosoritide

Ascendis PharmaTransCon CNP

Showing 15 of 28 trials with date data

Clinical Trials (28)

Total enrollment: 4,649 patients across 28 trials

NCT06164951 BridgeBio 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

NCT03424018 BioMarin 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

NCT03197766 BioMarin 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

NCT07441876 BioMarin PharmaceuticalBMN 333

A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

Start: Feb 2026Est. completion: Sep 2029160 patients

Phase 2/3Not Yet Recruiting

NCT05929807 Ascendis 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

NCT05598320 Ascendis 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

NCT07169279 BridgeBio 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

NCT06732895 Ascendis PharmaNavepegritide

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

Start: Dec 2024Est. completion: Jan 202724 patients

Phase 2Recruiting

NCT06433557 Ascendis PharmaCombination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections

A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia

Start: Jul 2024Est. completion: Nov 202722 patients

Phase 2Active Not Recruiting

NCT06079398 Ascendis 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

NCT05246033 Ascendis 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

NCT05145010 BridgeBio PharmaInfigratinib

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

Start: Dec 2021Est. completion: Feb 2032300 patients

Phase 2Enrolling By Invitation

NCT04638153 PfizerRecifercept

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

Start: Dec 2020Est. completion: Mar 202360 patients

Phase 2Terminated

NCT04554940 BioMarin 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

NCT04085523 Ascendis 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

NCT04265651 BridgeBio PharmaInfigratinib 0.016 mg/kg

Study of Infigratinib in Children With Achondroplasia

Start: Mar 2020Est. completion: Oct 202484 patients

Phase 2Completed

NCT03583697 BioMarin 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

NCT02724228 BioMarin 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

NCT02055157 BioMarin 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

NCT05813314 BioMarin 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

NCT01590446 BioMarin 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

NCT06168201 BioMarin 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

NCT05659719 PfizerRecifercept

A Study to Learn About Recifercept in Patients With Achondroplasia

Start: Oct 2022Est. completion: May 2023248 patients

N/ACompleted

NCT04035811 BridgeBio PharmaProspective Clinical Assessment Study in Children With Achondroplasia (ACH)

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Start: Aug 2019Est. completion: Jun 2026271 patients

N/AActive Not Recruiting

NCT03872531 BioMarin PharmaceuticalLifetime Impact Study for Achondroplasia

Lifetime Impact Study for Achondroplasia

Start: Mar 2019Est. completion: Feb 2021173 patients

N/ACompleted

NCT03449368 BioMarin 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

NCT02597881 BioMarin PharmaceuticalAchondroplasia Natural History Multicenter Clinical Study

Achondroplasia Natural History Multicenter Clinical Study

Start: Apr 2016Est. completion: Dec 20301,500 patients

N/ASuspended

NCT01603095 BioMarin 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

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

3 late-stage (Phase 3) programs — potential near-term approvals

4 actively recruiting trials targeting 4,649 patients

4 companies competing in this space