Achondroplasia

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At a Glance

Live from database

1FDA treatments

17Recruiting trials

Regulatory Watchboard

10 events

Feb 2026YUVIWEL: FDA approved

FDAcompleted

Feb 2026Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia

SYSNAV

TrialRECRUITING

Feb 2026A Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

BioMarin Pharmaceutical — PHASE2, PHASE3

TrialNOT YET RECRUITING

Dec 2025A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia

Abbisko Therapeutics Co, Ltd — PHASE1, PHASE2

TrialNOT YET RECRUITING

Nov 2025Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

QED Therapeutics, a BridgeBio company — PHASE2

TrialRECRUITING

Jun 2025A Study in Children With Achondroplasia

Abbisko Therapeutics Co, Ltd

TrialRECRUITING

Mar 2025A Study of TYRA-300 in Children With Achondroplasia: BEACH301

Tyra Biosciences, Inc — PHASE2

TrialRECRUITING

Dec 2024A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.

Ascendis Pharma A/S — PHASE2

TrialRECRUITING

Dec 2024Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）

Kyowa Kirin Co., Ltd. — PHASE3

TrialRECRUITING

Jul 2024A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia

Ascendis Pharma Growth Disorders A/S — PHASE2

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

4 programs

FINANCIAL LANDSCAPE SUMMARY

4

Total programs

4

Open now

3

Copay cards

1

Travel grants

Copay Assistance3

Voxzogo 0.4Mg

BioMarin Pharmaceutical Inc.

OpenContact for detailsApply ↗

VOXZOGO 0.4mg, VOXZOGO 0.56mg, VOXZOGO 1.2mg

BioMarin

OpenContact for detailsApply ↗

Yuviwel

Ascendis Pharma Growth Disorders A/S

OpenContact for details

Travel Grants1

BridgeBio Grants and Funding

OpenContact for detailsApply ↗

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Approved Treatments

1 FDA-approved

Voxzogo 0.4Mg

(VOSORITIDE)Orphan drugaccelerated

BioMarin Pharmaceutical Inc.

12.1 Mechanism of Action In patients with achondroplasia, endochondral bone growth is negatively regulated due to a gain of function mutation in fibro...

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Clinical Trial Landscape

17 active trials

2Phase 3

9Phase 2

5Unknown

1PHASE2, PHASE3

17Total recruiting

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Recent News & Research

No recent news articles indexed yet for Achondroplasia.
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Specialist Network

Top 6 by expertise

Doug Warner

Tyra Biosciences, Inc

Score 02 trials

Vegard Strom

Sunnaas Rehabilitation Hospital

Shuqi Zeng

12B, Building 1, No 515, Huanke Road, Pudong New Area, Shanghai 201210, China

Aimee D. Shu, M.D

Ascendis Pharma, Inc.

📍 STANFORD, CA

Vibeke Breinholt

Ascendis Pharma

Feihong Luo

Children's Hospital of Fudan University

Score 02 trials

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