VivoSeq
Other

Achondroplasia

15

Pipeline Programs↗

15

Active Trials↗

14

Companies Involved↗

Achondroplasia is an emerging landscape with 15 programs across 14 companies. The most active players are Tyra Biosciences, Inc, QED Therapeutics, a BridgeBio company, Ascendis Pharma A/S. 1 therapy is approved with 2 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

47/100

Pipeline Velocity

15/100

Mech. Concentration

50/100

Completion Rate

80/100

Attrition Rate

7/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

T

Tyra Biosciences, Inc

5 programs

Phase 2Q

QED Therapeutics, a BridgeBio company

4 programs

Phase 2A

Ascendis Pharma A/S

1 program

Phase 2A

Ascendis Pharma Growth Disorders A/S

1 program

Phase 2Pfizer logo

Pfizer

1 program

Phase 2A

Abbisko Therapeutics Co, Ltd

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

81%

Small molecule

13 of 16 classified programs — the dominant modality here.

19%ALL OTHERSPeptide213%Protein / enzyme16%

Small molecule leads; the ring shows all 16 classified programs, each share of the total. Modality classified for 70% of 23 pipeline programs.

Analysis

Trial Status

27

clinical trials

Completed8 · 30%
Recruiting7 · 26%
Active, not recruiting5 · 19%
Unknown status2 · 7%
Terminated2 · 7%
Enrolling by invitation2 · 7%
Other1 · 4%

Clinical Trials

Trial Activity

27 trials

Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
Recruiting·Kyowa Kirin Co., Ltd. logoKyowa Kirin Co., Ltd.·Phase 3·NCT06926491·6 enrolled
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Recruiting·AAscendis Pharma A/S·Phase 2·NCT06732895·24 enrolled
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
Recruiting·AAscendis Pharma A/S·Phase 2·NCT06079398·72 enrolled
Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children With Achondroplasia
Recruiting·QQED Therapeutics, a BridgeBio company·Phase 2·NCT07169279·77 enrolled
A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301
Recruiting·TTyra Biosciences, Inc·Phase 2·NCT06842355·92 enrolled
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Preclinical·NCT07441876·160 enrolled
VIrtual STudy in Achondroplasia for the US (VISTA)
Recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·N/A·NCT06168201·170 enrolled
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 2·NCT02724228·30 enrolled
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 2·NCT03989947·73 enrolled
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 3·NCT03424018·119 enrolled
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 2·NCT04554940·20 enrolled
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia
Active, not recruiting·AAscendis Pharma Growth Disorders A/S·Phase 2·NCT06433557·22 enrolled
A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
Not yet recruiting·AAbbisko Therapeutics Co, Ltd·Phase 1/2·NCT07297875·110 enrolled
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
Enrolling by invitation·QQED Therapeutics, a BridgeBio company·Phase 2·NCT05145010·300 enrolled
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
Enrolling by invitation·AAscendis Pharma Growth Disorders A/S·Phase 2/3·NCT05929807·140 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)