Condition (MeSH)
Achondroplasia
ClinicalTrials.gov lists 46 trials for Achondroplasia or a narrower condition beneath it. BioMarin Pharmaceutical has the most among industry and government lead sponsors, with 14.
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Trials per phase
46 of the 46 trials are tagged with Achondroplasia itself; the rest are tagged with a narrower condition beneath it.
When did Achondroplasia trials start? (15)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Observational
Top of scale: 5
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 2 |
| 2025 | 4 |
| 2024 | 4 |
| 2023 | 5 |
| 2022 | 4 |
| 2021 | 3 |
| 2020 | 5 |
| 2019 | 4 |
| 2018 | 2 |
| 2017 | 3 |
Showing 10 of 15 earlier years.
No forecast is made for this condition: it averages 4.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 8 trials still running at the 2026-10-09 snapshot; registered median of 27 months across 25 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Achondroplasia trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 1 |
| Phase 2 | 16 |
| Phase 2/3 | 3 |
| Phase 3 | 4 |
| Phase 4 | 1 |
| Observational | 19 |
How many Achondroplasia trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 10 |
| Enrolling by invitation | 2 |
| Active, not recruiting | 6 |
| Completed | 21 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Achondroplasia trials? (11)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| BioMarin Pharmaceutical | 14 |
| QED Therapeutics, a BridgeBio company | 5 |
| Ascendis Pharma A/S | 4 |
| Pfizer | 4 |
| Ascendis Pharma Growth Disorders A/S | 3 |
Showing 5 of 11 sponsors.
Which drugs are being tested for Achondroplasia? (4)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| BMN 111 | 7 |
| Navepegritide | 6 |
| Recifercept | 3 |
| Infigratinib | 2 |
Interventions as named in the registry (11)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| somatropin AQ | 3 |
| No Interventions | 1 |
| Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. | 1 |
| Infigratinib is provided as sprinkle capsules for daily oral administration | 1 |
| Vosoritide Injection [Voxzogo] | 1 |
Showing 5 of 11 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Achondroplasia trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Sleep apnoea syndrome | 4 | 3 |
| Influenza | 3 | 2 |
| Tonsillar hypertrophy | 3 | 2 |
| Adenoidal hypertrophy | 2 | 1 |
| Thyroglossal cyst | 2 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Achondroplasia? (26)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. | JAMA pediatrics | 2026 | Yes |
| Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. | Med (New York, N.Y.) | 2025 | Yes |
| Oral Infigratinib Therapy in Children with Achondroplasia. | The New England journal of medicine | 2025 | Yes |
| Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. | Clinical pharmacokinetics | 2024 | Yes |
| Natural disease course modeling of achondroplasia to evaluate the efficacy of recifercept in the absence of a placebo control arm in phase II study. | CPT: pharmacometrics & systems pharmacology | 2024 | Yes |
Showing 5 of 26 publications.
Prevalence (93)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Achondroplasia | Prevalence at birth | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Achondroplasia | Prevalence at birth | 1-9 / 100 000 | United Kingdom | GB | Unspecified |
| Achondroplasia | Prevalence at birth | 1-9 / 100 000 | United Kingdom | GB | Exact |
| Achondroplasia | Prevalence at birth | 1-5 / 10 000 | United Arab Emirates | Not stated | Unspecified |
| Achondroplasia | Prevalence at birth | 1-9 / 100 000 | Denmark | Not stated | Exact |
Showing 5 of 93 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Achondroplasia | 15 | Exact | Orphadata Science knowledge files, Product 1 |
| Achondroplasia | 15 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.