Disease

Achondroplasia

Late-stage therapeutic development

Also known as ACH, achondroplastic physique, chondrodystrophia, achondroplastic dwarfism.

Clinical trials

Associated genes

Related proteins

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this disease.

Approval: Voxzogo (EMA)

Regulatory2021-08-26EMA

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate

Key developments

5 clinical trials expected to report results, the earliest in Q2 2029.

Upcoming Milestones

Major developments

Upcoming trial readoutHigh impact

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

Results expected Q2 20292026-07-06

Major trial resultsHigh impact

ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period

Results posted2026-07-02

Clinical Milestones7View

Results posted 1

2026-07-02ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension PeriodResults posted

Results expected 5

2026-07-13Phase 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 AchondroplasiaResults expected Q1 2030
2026-07-06A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaResults expected Q2 2029
2026-06-30A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301Results expected Q1 2030
2026-03-13A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With AchondroplasiaResults expected Q2 2038
2026-03-13A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With AchondroplasiaResults expected Q2 2031

Recently completed 1

2025-12-18A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age With Achondroplasia: PROPEL 3Completed

Activity timeline7

2026-07-13ClinicalPhase 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 AchondroplasiaResults expected Q1 2030
2026-07-06ClinicalA Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaResults expected Q2 2029
2026-07-02ClinicalACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension PeriodResults posted
2026-06-30ClinicalA Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301Results expected Q1 2030
2026-03-13ClinicalA Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With AchondroplasiaResults expected Q2 2038
2026-03-13ClinicalA Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With AchondroplasiaResults expected Q2 2031
2025-12-18ClinicalA Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age With Achondroplasia: PROPEL 3Completed

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies

Vosoritideapproved

Approval — Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and… (2021)

Clinical trials

7 sponsors · 2 new · 1 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Late-stage studies

A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia

Phase 3 · Active, not recruiting · BioMarin Pharmaceutical

A Multicenter, Open-label, Single-arm, Phase 3 Study of KK8398 (Infigratinib) in Patients With Achondroplasia (AOBA Study)

Phase 3 · Recruiting · Kyowa Kirin Co., Ltd.

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

Phase 2/3 · Recruiting · BioMarin Pharmaceutical

A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age With Achondroplasia: PROPEL 3

Phase 3 · Completed · QED Therapeutics, a BridgeBio company

A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children With Achondroplasia Followed by an Open Label Extension Period

Phase 2/3 · Completed · Ascendis Pharma Growth Disorders A/S

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Phase 3 · Completed · BioMarin Pharmaceutical

Recruiting

A Multicenter, Open-label, Single-arm, Phase 3 Study of KK8398 (Infigratinib) in Patients With Achondroplasia (AOBA Study)

Phase 3 · Recruiting · Kyowa Kirin Co., Ltd.

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

Phase 2/3 · Recruiting · BioMarin Pharmaceutical

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

Phase 2 · Recruiting · QED Therapeutics, a BridgeBio company

A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301

Phase 2 · Recruiting · Tyra Biosciences, Inc

Recently completed

A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age With Achondroplasia: PROPEL 3

Phase 3 · Completed · QED Therapeutics, a BridgeBio company

Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2

Phase 2 · Completed · QED Therapeutics, a BridgeBio company

Phase 2/3 · Completed · Ascendis Pharma Growth Disorders A/S

Phase 2 · Completed · Ascendis Pharma A/S

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Phase 3 · Completed · BioMarin Pharmaceutical

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals

2021emaApproval — Vosoritide· Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

4 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

FGFR30.82 NPR20.57 CNP0.13 TAS2R140.01

Disease biology

2 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Fibroblast growth factor receptor 3via FGFR30.82 Atrial natriuretic peptide receptor 2via NPR20.57

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

An autosomal dominant disorder that is the most frequent form of short-limb dwarfism. Affected individuals exhibit short stature caused by rhizomelic shortening of the limbs, characteristic facies with frontal bossing and mid-face hypoplasia, exaggerated lumbar lordosis, limitation of elbow extension, GENU VARUM, and trident hand. (Online Mendelian Inheritance in Man, http://www.ncbi.nlm.nih.gov/Omim, MIM#100800, April 20, 2001)

Synonyms

ACH, achondroplastic physique, chondrodystrophia, achondroplastic dwarfism

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
Related entities are derived from literature co-mention (studied together) — associative, not causal.